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| dmd gene therapy approval: Muscle Gene Therapy Dongsheng Duan, 2009-11-26 Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy. |
| dmd gene therapy approval: Exploring Novel Clinical Trial Designs for Gene-Based Therapies National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Forum on Regenerative Medicine, 2020-08-27 Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop. |
| dmd gene therapy approval: Neuromuscular Disorders: Management and Treatment E-Book Tulio E. Bertorini, 2010-09-08 Neuromuscular Disorders presents a multi-disciplinary approach to the management and therapeutic treatment of the full range of neuromuscular disorders and resulting complications. Dr. Tulio Bertorini and a contributing team of the world’s leading authorities in the field provide the latest tools and strategies for minimizing disability and maximizing quality of life. Effectively treat your patients using the latest management tools and targeted therapeutic strategies. Manage all neuromuscular disorders as well as resulting complications through comprehensive coverage of diagnosis and evaluations, treatments, and outcomes. Apply the multi-disciplinary approach of an expert in clinical neuromuscular care and a team of world-renown contributors. Easily refer to tools for diagnosis, treatment algorithms, and drug tables included throughout the text. |
| dmd gene therapy approval: Regulatory Aspects of Gene Therapy and Cell Therapy Products Maria Cristina Galli, Mercedes Serabian, 2015-09-15 This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent must-know guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals. |
| dmd gene therapy approval: Neuromuscular Disorders of Infancy, Childhood, and Adolescence Basil T. Darras, H. Royden Jones Jr., Monique M. Ryan, Darryl C. De Vivo, 2014-12-03 Neuromuscular disorders are diagnosed across the lifespan and create many challenges especially with infants, children and adolescents. This new edition of the definitive reference, edited by the established world renowned authorities on the science, diagnosis and treatment of neuromuscular disorders in childhood is a timely and needed resource for all clinicians and researchers studying neuromuscular disorders, especially in childhood. The Second Edition is completely revised to remain current with advances in the field and to insure this remains the standard reference for clinical neurologists and clinical research neurologists. The Second Edition retains comprehensive coverage while shortening the total chapter count to be an even more manageable and effective reference. - Carefully revised new edition of the classic reference on neuromuscular disorders in infancy, childhood and adolescence. - Definitive coverage of the basic science of neuromuscular disease and the latest diagnosis and treatment best practices. - Includes coverage of clinical phenomenology, electrophysiology, histopathology, molecular genetics and protein chemistry |
| dmd gene therapy approval: Rare Disease Drug Development Raymond A. Huml, 2021-11-08 This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers. |
| dmd gene therapy approval: Introduction to Basics of Pharmacology and Toxicology Gerard Marshall Raj, Ramasamy Raveendran, 2019-11-16 This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology. |
| dmd gene therapy approval: Muscle Gene Therapy Dongsheng Duan, Jerry R. Mendell, 2019-03-30 About 7 million people worldwide are suffering from various inherited neuromuscular diseases. Gene therapy brings the hope of treating these diseases at their genetic roots. Muscle Gene Therapy is the only book dedicated to this topic. The first edition was published in 2010 when the field was just about to enter its prime time. The progress made since then has been unprecedented. The number of diseases that have been targeted by gene therapy has increased tremendously. The gene therapy toolbox is expanded greatly with many creative novel strategies (such as genome editing and therapy with disease-modifying genes). Most importantly, clinical benefits have begun to emerge in human patients. To reflect rapid advances in the field, we have compiled the second edition of Muscle Gene Therapy with contributions from experts that have conducted gene therapy studies either in animal models and/or in human patients. The new edition offers a much needed, up-to-date overview and perspective on the foundation and current status of neuromuscular disease gene therapy. It provides a framework to the development and regulatory approval of muscle gene therapy drugs in the upcoming years. This book is a must-have for anyone who is interested in neuromuscular disease gene therapy including those in the research arena (established investigators and trainees in the fields of clinical practice, veterinary medicine and basic biomedical sciences), funding and regulatory agencies, and patient community. |
| dmd gene therapy approval: Innovation in Clinical Trial Methodologies Peter Schueler, 2020-11-20 Innovation in Clinical Trial Methodologies: Lessons Learned during the Corona Pandemic presents a selection of updated chapters from Re-Engineering Clinical Trials that feature innovative options and methods in clinical trials. The Coronavirus pandemic is an accelerator for digitalization in many industries, including clinical trials. This book considers best practices, alternative study concepts requiring fewer patients, studies with less patient interaction, the design of virtualized protocols, and moving from data to decisions. This book will be helpful to pharmacologists, physicians and clinical researchers involved in the process of clinical development and clinical trial design. Considers multiple digital and virtual strategies Explores best practices, including the use of reduced patient involvement Brings together expert, trusted information to increase the efficiency and effectiveness of clinical trials |
| dmd gene therapy approval: Translating Regenerative Medicine to the Clinic Jeffrey Laurence, Pedro Baptista, Anthony Atala, 2015-11-18 Translating Regenerative Medicine to the Clinic reviews the current methodological tools and experimental approaches used by leading translational researchers, discussing the uses of regenerative medicine for different disease treatment areas, including cardiovascular disease, muscle regeneration, and regeneration of the bone and skin. Pedagogically, the book concentrates on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field. It promotes cross-disciplinary communication between the sub-specialties of medicine, but remains unified in theme by emphasizing recent innovations, critical barriers to progress, the new tools that are being used to overcome them, and specific areas of research that require additional study to advance the field as a whole. Volumes in the series include Translating Gene Therapy to the Clinic, Translating Regenerative Medicine to the Clinic, Translating MicroRNAs to the Clinic, Translating Biomarkers to the Clinic, and Translating Epigenetics to the Clinic. - Encompasses the latest innovations and tools being used to develop regenerative medicine in the lab and clinic - Covers the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field - Contains extensive pedagogical updates aiming to improve the education of translational researchers in this field - Provides a transdisciplinary approach that supports cross-fertilization between different sub-specialties of medicine |
| dmd gene therapy approval: Neurology and Pregnancy Eric A.P. Steegers, Marilyn J. Cipolla, Eliza C. Miller, 2020-08-05 Neurology and Pregnancy provides a comprehensive multidisciplinary guide to best practices for research and practicing neurologists, as well as obstetricians and other specialists caring for women with an acute or chronic neurological disorder. The book encompasses preconception care, genetic counseling, pregnancy in patients with chronic neurological disorders, and acute pregnancy-related neurological complications. Postpartum care and complications, including lactation concerns, are also addressed, as well as the long-term effects of pregnancy and its complications on maternal brain health. Vol 1 summarizes the complex neurophysiological changes in pregnancy from a basic and translational science perspective. This includes neuroimaging, principles of neuro-obstetric critical care, and ethical and medicolegal concerns, describes normal fetal cerebral development and summarizes the management of the most common prenatal neurological diagnoses. Vol 2 focuses on chronic neurological conditions in pregnancy such as epilepsy, migraine, and multiple sclerosis, as well as acute neurological disorders including preeclampsia/ eclampsia, ischemic and hemorrhagic stroke, and CNS infections. Each chapter introduction includes data on epidemiology, when applicable. In discussing management, comments of safety profiles of medications preconceptionally, during pregnancy and lactation are summarized. Each chapter includes 1-2 illustrative cases. - Encompasses both chronic and acute neurological disorders in pregnancy - Includes epilepsy, sleep disorders, MS, headaches, neuromuscular disorders - Covers spinal injury and movement disorders - Contains cerebrovascular diseases and brain tumors - Examines stroke, hemorrhage, TBI, preeclampsia and more - Supplies illustrative case study examples |
| dmd gene therapy approval: Gene Therapy of the Central Nervous System: From Bench to Bedside Michael G. Kaplitt, Matthew During, 2006 Few areas of biomedical research provide greater opportunities to capitalize upon the revolution in genomics and molecular biology than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy in the Brain: From Bench to Bedside represents the definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped to create the field as well as those who are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects which would be of interest to anyone who is currently using or contemplating exploring gene therapy for neurobiological applications. Basic gene transfer technologies are discussed, with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells. Numerous research applications are reviewed, particularly in complex fields such as behavioral neurobiology. Several preclinical areas are also covered which are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis. Among the most exciting advances in recent years has been the use of neurological gene therapy in human clinical trials, including Parkinson's disease, Canavan disease and Batten disease. Finally, readers will find insider information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use. This work provides up-to-date information and key insights into those gene therapy issues which are important to both scientists and clinicians focusing upon the brain and central nervous system. |
| dmd gene therapy approval: Human Genome Editing National Academies of Sciences, Engineering, and Medicine, National Academy of Medicine, National Academy of Sciences, Committee on Human Gene Editing: Scientific, Medical, and Ethical Considerations, 2017-08-13 Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing. |
| dmd gene therapy approval: A Handbook of Gene and Cell Therapy Clévio Nóbrega, Liliana Mendonça, Carlos A. Matos, 2020-06-27 This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field. |
| dmd gene therapy approval: Drug Development for Gene Therapy Yanmei Lu, Boris Gorovits, 2024-02-28 Drug Development for Gene Therapy Industry-centric perspective on translational and bioanalytical challenges and best practices for gene therapies Drug Development for Gene Therapy focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities, presenting a significant body of data, including information related to safety and efficacy, necessary to advance through the development pipeline into clinical use. The text covers bioanalytical methods and platforms including patient screening assays, different PCR tests, enzyme activity assays, ELISpot, NGS, LC/MS, and immunoassays, with FDA and EMA guidelines on gene therapy safety and efficacy, along with companion diagnostics regulations from US and EU perspectives. The chapters offer an in-depth discussion of the basics and best practices for translational biomarkers, bioanalysis, and developing companion diagnostics / lab tests for gene therapies in the pharma and biopharma industries. To aid in reader comprehension, the text includes clinical examples of relevant therapies in related chapters. Some of the core topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues. Written by two highly qualified authors with significant experience in the field, Drug Development for Gene Therapy includes information on: Bioanalytical methods to detect pre-existing antibodies against adeno-associated viruses (AAV) capsids Detection of cellular immunity and humoral response to viral capsids and transgene proteins, and immunogenicity of gene therapy products Nonclinical and clinical study considerations and methods for biodistribution and shedding Quantification of transgene protein expression and biochemical function, and substrate and distal pharmacodynamic biomarker measurements for gene therapy Detection and quantification of rAAV integration and off-target editing Current regulatory landscape for gene therapy product development and the role of biomarkers and general regulatory considerations for gene therapy companion diagnostics With comprehensive coverage of the subject, Drug Development for Gene Therapy is a must-have resource for researchers and developers in the areas of pharmaceuticals, biopharmaceuticals, and contract research organizations (CROs), along with professors, researchers, and advanced students in chemistry, biological, biomedical engineering, pharmaceuticals, and medical sciences. |
| dmd gene therapy approval: The Human Genome Julia E. Richards, R. Scott Hawley, 2010-12-12 Significant advances in our knowledge of genetics were made during the twentieth century but in the most recent decades, genetic research has dramatically increased its impact throughout society. Genetic issues are now playing a large role in health and public policy, and new knowledge in this field will continue to have significant implications for individuals and society. Written for the non-majors human genetics course, Human Genetics, Third Edition will increase the genetics knowledge of students who are learning about human genetics for the first time. This thorough revision of the best-selling Human Genome, Second Edition includes entirely new chapters on forensics, stem cell biology, bioinformatics, and societal/ethical issues associated with the field. New special features boxes make connections between human genetics and human health and disease. Carefully crafted pedagogy includes chapter-opening case studies that set the stage for each chapter; concept statements interspersed throughout the chapter that keep first-time students focused on key concepts; and end-of-chapter questions and critical thinking activities. This new edition will contribute to creating a genetically literate student population that understands basic biological research, understands elements of the personal and health implications of genetics, and participates effectively in public policy issues involving genetic information. - Includes topical material on forensics, disease studies, and the human genome project to engage non-specialist students - Full, 4-color illustration program enhances and reinforces key concepts and themes - Uniform organization of chapters includes interest boxes that focus on human health and disease, chapter-opening case studies, and concept statements to engage non-specialist readers |
| dmd gene therapy approval: Heritable Human Genome Editing The Royal Society, National Academy of Sciences, National Academy of Medicine, International Commission on the Clinical Use of Human Germline Genome Editing, 2021-01-16 Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight. |
| dmd gene therapy approval: Muscular Dystrophy Therapeutics Rika Maruyama, Toshifumi Yokota, 2022-11-18 This detailed book presents a comprehensive collection of state-of-the-art protocols on muscular dystrophy therapeutics, covering therapeutics using antisense oligonucleotides, gene replacement, genome editing, small molecules, stem cells, and antibodies. Written by leaders in the field, the volume explores techniques that are currently in use and are starting an exciting therapeutic revolution in muscular dystrophy. As a part of the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step and readily reproducible laboratory protocols, as well as tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Muscular Dystrophy Therapeutics: Methods and Protocols serves as an ideal resource to inspire readers and provide tips, strategies, and advice to develop new therapeutic technologies for this group of diseases. |
| dmd gene therapy approval: Gahart's 2021 Intravenous Medications - E-Book Betty L. Gahart, Adrienne R. Nazareno, Meghan Ortega RN, 2020-06-02 The essential information you need to safely administer more than 400 intravenous drugs! For over 45 years, Gahart’s 2021 Intravenous Medications: A Handbook for Nurses and Health Professionals has been a trusted resource for comprehensive drug coverage, unparalleled accuracy, and an intuitive quick-access format. In addition to updated drug interactions, precautions, alerts, and patient teaching instructions for all existing IV drugs, this new 37th edition includes more than a dozen new monographs of the most recent IV drugs to be approved by the FDA. Administering intravenous drugs is a critical task — inaccurate or out-of-date information is not an option. Known as the #1 IV drug handbook on the market, Gahart’s annual publication, with its history of impeccable accuracy, gives you the extra confidence and guidance you need to safely and effectively treat patients. Monographs on more than 400 IV drugs offers an impressive breadth of coverage that goes well beyond any comparable drug reference. Updated annual publication prevents you from referencing outdated information. Additional drug monographs are housed on the companion Evolve website. A 45-year history of impeccable accuracy reinforces the importance of safe IV drug administration. The perfect depth of information equips you with everything that is needed by today’s clinicians for safe administration of IV drugs — nothing more, nothing less. Proven, clinically optimized format keeps all dosage information for each drug on either a single page or a two-page spread to prevent hand contamination by having to turn a page. Highlighted Black Box Warnings and relevant content make locating critical information fast and easy. Special circumstances in blue-screened text call attention to important circumstances that may not warrant Black Box Warnings. Life stage dosage variances are highlighted for geriatric, pediatric, infant, and neonatal patients. Dilution and dosage charts within monographs provide quick access to essential clinical information. Convenient, alphabetical format organizes all drug monographs by generic name, allowing you to find any drug in seconds. NEW! Drug monographs for 19 newly approved drugs by the FDA provides you with the most current drug information. Updates on drug interactions, precautions, alerts, and more have been made throughout the guide to reflect all changes to existing medications. |
| dmd gene therapy approval: Animal Models in Medicine and Biology Eva Tvrdá, Sarat Chandra Yenisetti, 2020-04-08 Thanks to animal models, our knowledge of biology and medicine has increased enormously over the past decades, leading to significant breakthroughs that have had a direct impact on the prevention, management and treatment of a wide array of diseases.This book presents a comprehensive reference that reflects the latest scientific research being done in a variety of medical and biological fields utilizing animal models. Chapters on Drosophila, rat, pig, rabbit, and other animal models reflect frontier research in neurology, psychiatry, cardiology, musculoskeletal disorders, reproduction, chronic diseases, epidemiology, and pain and inflammation management. Animal Models in Medicine and Biology offers scientists, clinicians, researchers and students invaluable insights into a wide range of issues at the forefront of medical and biological progress. |
| dmd gene therapy approval: Platform Technologies in Drug Discovery and Validation , 2017-11-21 Platform Technologies in Drug Discovery and Validation, Volume 50, the latest release in the Annual Reports in Medicinal Chemistry series, provides timely and critical reviews of important topics in medicinal chemistry, with an emphasis on emerging topics in the biological sciences. Topics covered in this new volume include DELT, Oligos: ASO, siRNA, CRISPR, Micro-fluidic chemistry, High throughput screening, Kinase-centric computational drug development, Virtual Screening, Phenotypic screening, PROTACS, Chemical Biology, Fragment-based lead generation, Antibody-Drug Conjugates, Antibody-recruiting small molecules, Deuteration, and Peptides. - Unique for its treatment of platform technologies for medicinal chemistry and target validation - Provides a single, rich volume that summaries a broad spectrum of expertise relevant to the field - Presents state-of-the-art summaries of platform technologies |
| dmd gene therapy approval: A Guide to Human Gene Therapy Roland W. Herzog, Sergei Zolotukhin, 2010 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne |
| dmd gene therapy approval: The Fourth Industrial Revolution Klaus Schwab, 2017-01-03 The founder and executive chairman of the World Economic Forum on how the impending technological revolution will change our lives We are on the brink of the Fourth Industrial Revolution. And this one will be unlike any other in human history. Characterized by new technologies fusing the physical, digital and biological worlds, the Fourth Industrial Revolution will impact all disciplines, economies and industries - and it will do so at an unprecedented rate. World Economic Forum data predicts that by 2025 we will see: commercial use of nanomaterials 200 times stronger than steel and a million times thinner than human hair; the first transplant of a 3D-printed liver; 10% of all cars on US roads being driverless; and much more besides. In The Fourth Industrial Revolution, Schwab outlines the key technologies driving this revolution, discusses the major impacts on governments, businesses, civil society and individuals, and offers bold ideas for what can be done to shape a better future for all. |
| dmd gene therapy approval: Exon Skipping and Inclusion Therapies Toshifumi Yokota, Rika Maruyama, 2018-09-01 This book presents a comprehensive collection of detailed state-of-the-art exon skipping and splices modulation protocols. Chapters detail 14 genetic diseases, AON-mediated therapies, and CRISPR/Cas9-mediated gene editing therapies. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help researchers initiate the development of next-generation therapies. |
| dmd gene therapy approval: Development of Gene Therapies Avery McIntosh, Oleksandr Sverdlov, 2024-05-23 Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx Insights into commercial models, access hurdles, and health economics of gene therapies Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective |
| dmd gene therapy approval: Cell and Gene Therapies Miguel-Angel Perales, Syed A. Abutalib, Catherine Bollard, 2018-11-27 In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee. |
| dmd gene therapy approval: Translational Bioinformatics in Healthcare and Medicine , 2021-05-13 Translational Bioinformatics in Healthcare and Medicine offers an overview of main principles of bioinformatics, biological databases, clinical informatics, health informatics, viroinformatics and real-case applications of translational bioinformatics in healthcare. Written by experts from both technology and clinical sides, the content brings together essential knowledge to make the best of recent advancements of the field. The book discusses topics such as next generation sequence analysis, genomics in clinical care, IoT applications, blockchain technology, patient centered interoperability of EHR, health data mining, and translational bioinformatics methods for drug discovery and drug repurposing. In addition, it discusses the role of bioinformatics in cancer research and viroinformatics approaches to counter viral diseases through informatics. This is a valuable resource for bioinformaticians, clinicians, healthcare professionals, graduate students and several members of biomedical field who are interested in learning more about how bioinformatics can impact in their research and practice. - Covers recent advancements in translational bioinformatics and its healthcare applications - Discusses integrative and multidisciplinary approaches to U-healthcare systems development and management - Bridges the gap among various knowledge domains in the field, integrating both technological and clinical knowledge into practical content |
| dmd gene therapy approval: NORD Guide to Rare Disorders National Organization for Rare Disorders, 2003 NORD Guide to Rare Disorders is a comprehensive, practical, authoritative guide to the diagnosis and management of more than 800 rare diseases. The diseases are discussed in a uniform, easy-to-follow format--a brief description, signs and symptoms, etiology, related disorders, epidemiology, standard treatment, investigational treatment, resources, and references.The book includes a complete directory of orphan drugs, a full-color atlas of visual diagnostic signs, and a Master Resource List of support groups and helpful organizations. An index of symptoms and key words offers physicians valuable assistance in finding the information they need quickly. |
| dmd gene therapy approval: Practical Approach to the Neurological Patient - E-BOOK William J. Mullally, 2024-07-25 To the non-neurologist, neurology can be one of the most intimidating fields of medicine, yet it includes many common problems faced in everyday primary care practice. Written specifically for the general clinician, Practical Approach to the Neurological Patient: A Clinician's Guide provides clear, up-to-date, and easy-to-understand guidance on commonly encountered issues, helping you take an informed approach to patients with neurological concerns. Dr. William J. Mullally and a team of expert contributing authors address headache, dizziness, stroke, pain, head trauma, and much more, making this volume an indispensable resource for primary care practitioners, internists, family practitioners, medical specialists, medical residents, nurse practitioners, physician associates, and students. - Offers concise, comprehensive content designed to help guide the primary care provider or non-neurologist on how to manage patients with common neurological conditions. - Covers timely topics such as women's neurology, neurogenetics, pain neurology, sleep disorders, dementia, and headache (including facial pain). - Includes Key Points in every chapter, MRI images that show brain lesions, figures and tables throughout, and clinical algorithms to support everyday decision making. - Features multidisciplinary input from physician authors who are contributors to the American Journal of Medicine, as well as a nurse practitioner and physician associates. |
| dmd gene therapy approval: Curing Genetic Diseases through Genome Reprogramming , 2021-06-24 Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome. - Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing - Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects - Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations |
| dmd gene therapy approval: Ferri's Clinical Advisor 2025 - E-BOOK Fred F. Ferri, 2024-06-05 For more than 25 years, Ferri's Clinical Advisor has provided immediate answers on the myriad medical diseases and disorders you're likely to encounter in a unique, easy-to-use format. A bestselling title year after year, this popular 5 books in 1 reference delivers vast amounts of information in a user-friendly manner. It is updated annually to provide current and clinically relevant answers on over 1,000 common medical conditions, including diseases and disorders, differential diagnoses, clinical algorithms, laboratory tests, and clinical practice guidelines?all carefully reviewed by experts in key clinical fields. Extensive algorithms, along with hundreds of high-quality photographs, illustrations, diagrams, and tables, ensure that you stay current with today's medical practice. - Contains significant updates throughout all 5 sections, covering all aspects of diagnosis and treatment. - Features 26 all-new topics including eosinophilic gastroenteritis (EGE), retroperitoneal abscess, adenomyosis, periprosthetic joint infection, tonic pupil, rectal adenocarcinoma, nightmares and dream disturbances, avoidance/restrictive food intake disorder (ARFID), hypokalemia, cardiac implantable electronic device infection, heparin resistance, and hypertrophic pyloric stenosis, among others. - Includes useful appendices covering palliative care, preoperative evaluation, nutrition, poison management, commonly used herbal products in integrated medicine, and much more. - Offers online access to newly revised and updated Patient Teaching Guides in both English and Spanish. - Any additional digital ancillary content may publish up to 6 weeks following the publication date. |
| dmd gene therapy approval: Principles of Cloning Jose Cibelli, Ian Wilmut, Rudolf Jaenisch, John Gurdon, Robert Lanza, Michael West, Keith H.S. Campbell, 2013-09-24 Principles of Cloning, Second Edition is the fully revised edition of the authoritative book on the science of cloning. The book presents the basic biological mechanisms of how cloning works and progresses to discuss current and potential applications in basic biology, agriculture, biotechnology, and medicine. Beginning with the history and theory behind cloning, the book goes on to examine methods of micromanipulation, nuclear transfer, genetic modification, and pregnancy and neonatal care of cloned animals. The cloning of various species—including mice, sheep, cattle, and non-mammals—is considered as well. The Editors have been involved in a number of breakthroughs using cloning technique, including the first demonstration that cloning works in differentiated cells done by the Recipient of the 2012 Nobel Prize for Physiology or Medicine – Dr John Gurdon; the cloning of the first mammal from a somatic cell – Drs Keith Campbell and Ian Wilmut; the demonstration that cloning can reset the biological clock - Drs Michael West and Robert Lanza; the demonstration that a terminally differentiated cell can give rise to a whole new individual – Dr Rudolf Jaenisch and the cloning of the first transgenic bovine from a differentiated cell – Dr Jose Cibelli. The majority of the contributing authors are the principal investigators on each of the animal species cloned to date and are expertly qualified to present the state-of-the-art information in their respective areas. - First and most comprehensive book on animal cloning, 100% revised - Describes an in-depth analysis of current limitations of the technology and research areas to explore - Offers cloning applications on basic biology, agriculture, biotechnology, and medicine |
| dmd gene therapy approval: Pediatric Pharmacotherapy Wieland Kiess, Matthias Schwab, Johannes van den Anker, 2020-07-15 This volume provides readers with the most updated scientific information on the efficacy and safety of medicines for children and adolescents. The book enriches the understanding of pediatric pharmacotherapy for health professionals, regulatory agencies, pharmaceutical companies and learned societies. It contains important information on the pharmacodynamics and pharmacokinetics of drugs. It summarizes the latest investigations on the effects of pharmacological treatments in relation to and dependent on age, gender, fat mass and disease status. Therefore and importantly, this volume reviews the latest data on how pharmacotherapy has to be adjusted and personalized in regards to stages of development and during the pediatric lifespan from neonate through adolescence. In addition, the topic of rare diseases and special challenges for pharmacotherapy will be included and will provide readers with the necessary knowledge to handle complex diseases and treatment strategies especially in relation to pharmacotherapy of rare and orphan diseases. |
| dmd gene therapy approval: Gene and Cell Therapy Nancy Smyth Templeton, 2003-12-17 This reference is completely revised and expanded to reflect the most critical studies, controversies, and technologies impacting the medical field, including probing research on lentivirus, gutless adenovirus, bacterial and baculovirus vectors, retargeted viral vectors, in vivo electroporation, in vitro and in vivo gene detection systems, and all inducible gene expression systems. Scrutinizing every tool, technology, and issue impacting the future of gene and cell research, it is specifically written and organized for laymen, scholars, and specialists from varying backgrounds and disciplines to understand the current status of gene and cell therapy and anticipate future developments in the field. |
| dmd gene therapy approval: Lung Epithelial Biology in the Pathogenesis of Pulmonary Disease Venkataramana K Sidhaye, Michael Koval, 2017-03-09 Lung Epithelial Biology in the Pathogenesis of Pulmonary Disease provides a one-stop resource capturing developments in lung epithelial biology related to basic physiology, pathophysiology, and links to human disease. The book provides access to knowledge of molecular and cellular aspects of lung homeostasis and repair, including the molecular basis of lung epithelial intercellular communication and lung epithelial channels and transporters. Also included is coverage of lung epithelial biology as it relates to fluid balance, basic ion/fluid molecular processes, and human disease. Useful to physician and clinical scientists, the contents of this book compile the important and most current findings about the role of epithelial cells in lung disease. Medical and graduate students, postdoctoral and clinical fellows, as well as clinicians interested in the mechanistic basis for lung disease will benefit from the books examination of principles of lung epithelium functions in physiological condition. - Provides a single source of information on lung epithelial junctions and transporters - Discusses of the role of the epithelium in lung homeostasis and disease - Includes capsule summaries of main conclusions as well as highlights of future directions in the field - Covers the mechanistic basis for lung disease for a range of audiences |
| dmd gene therapy approval: Assessing Genetic Risks Institute of Medicine, Committee on Assessing Genetic Risks, 1994-01-01 Raising hopes for disease treatment and prevention, but also the specter of discrimination and designer genes, genetic testing is potentially one of the most socially explosive developments of our time. This book presents a current assessment of this rapidly evolving field, offering principles for actions and research and recommendations on key issues in genetic testing and screening. Advantages of early genetic knowledge are balanced with issues associated with such knowledge: availability of treatment, privacy and discrimination, personal decision-making, public health objectives, cost, and more. Among the important issues covered: Quality control in genetic testing. Appropriate roles for public agencies, private health practitioners, and laboratories. Value-neutral education and counseling for persons considering testing. Use of test results in insurance, employment, and other settings. |
| dmd gene therapy approval: Principles of Good Clinical Practice Michael J. McGraw, 2010 Part of RPS Pharmacy Business Administration Series, this book offers good clinical practice guidelines. It includes standards on how clinical trials should be conducted, provide assurance of safety and efficacy of various drugs and protect human rights. |
| dmd gene therapy approval: Duchenne Muscular Dystrophy Alan E. H. Emery, Francesco Muntoni, Rosaline C. M. Quinlivan, 2015-02-19 Duchenne Muscular Dystrophy, an inherited and progressive muscle wasting disease, is one of the most common single gene disorders found in the developed world. In this fourth edition of the classic monograph on the topic, Alan Emery and Francesco Muntoni are joined by Rosaline Quinlivan, Consultant in Neuromuscular Disorders, to provide a thorough update on all aspects of the disorder. Recent understanding of the nature of the genetic defect responsible for Duchenne Muscular Dystrophy and isolation of the protein dystrophin has led to the development of new theories for the disease's pathogenesis. This new edition incorporates these advances from the field of molecular biology, and describes the resultant opportunities for screening, prenatal diagnosis, genetic counselling and from recent pioneering work with anti-sense oligonucleotides, the possibility of effective RNA therapy. Although there is still no cure for the disorder, there have been significant developments concerning the gene basis, publication of standards of care guidelines, and improvements in management leading to significantly longer survival, particularly with cardio-pulmonary care. The authors also investigate other forms of pharmacological, cellular and gene therapies. Duchenne Muscular Dystrophy will be essential reading not only for scientists and clinicians, but will also appeal to therapists and other professionals involved in the care of patients with muscular dystrophy. |
| dmd gene therapy approval: Characterization and Clinical Management of Dilated Cardiomyopathy Marco Merlo, 2021-01-12 Dilated cardiomyopathy (DCM) is a particular phenotype of non-ischemic systolic heart failure, frequently recognizing a genetic background and affecting relatively young patients with few comorbidities. Nowadays, long-term survival of DCM patients has been markedly improved due to an early diagnosis and uninterrupted and tailored follow-up under constant optimal medical and non-pharmacological evidence-based treatments. Nevertheless, DCM is still one of the most common causes of heart transplantation in the western world. Clinical management requires an integrated and systematic use of diagnostic tools and a deeper investigation of the basic mechanisms underlying the disease. However, several emerging issues remain debated. Specifically, the genotype–phenotype correlation, the role of advanced imaging techniques and genetic testing, the lack of appropriate risk stratification models, the need for a multiparametric and multidisciplinary approach for device implantation, and a continuous reclassification of the disease during follow-up remain challenging issues in clinical practice. Therefore, the aim of this Special Issue is to shed the light on the most recent advancements in characterization and clinical management of DCM in order to unveil the conundrum of this particular disease. |
| dmd gene therapy approval: Cardiomyopathy Gustav Mattsson, Peter Magnusson, 2021 Cardiomyopathies are diseases of the heart muscle with diverse etiologies ranging from myocarditis to gene mutations. They are classified according to morphology and function, and then further categorized based on whether they are familial or non-familial and based on specific etiologies. This book examines the various cardiomyopathies, including arrhythmogenic cardiomyopathy, hypertrophic cardiomyopathy, and dilated cardiomyopathy, as well as their genetic basis. |
Duchenne Muscular Dystrophy (DMD): Symptoms & Treatment - Cleveland Clinic
Jul 25, 2022 · Duchenne muscular dystrophy (DMD) is a condition that causes skeletal and heart muscle weakness that quickly gets worse with time. Symptoms usually begin by the age of 6 …
Duchenne muscular dystrophy - Wikipedia
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. [3] [7] [8] The onset of muscle weakness typically begins around age four, with …
Duchenne Muscular Dystrophy - StatPearls - NCBI Bookshelf
Jul 10, 2023 · Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. It is the most common hereditary neuromuscular disease and does not …
About Duchenne Muscular Dystrophy - National Human …
Apr 18, 2013 · DMD is a rapidly progressive form of muscular dystrophy that occurs primarily in boys. It is caused by an alteration (mutation) in a gene, called the DMD gene that can be …
Duchenne Muscular Dystrophy - Johns Hopkins Medicine
Duchenne muscular dystrophy, or DMD, is associated with the most severe clinical symptoms of all the muscular dystrophies. It is caused by a genetic mutation on one of the mother’s X …
Duchenne Muscular Dystrophy: Symptoms, Treatment, and More
Jun 5, 2024 · Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disease that affects the muscles. Caused by a defective gene and first appearing in childhood, …
What To Know About Duchenne Muscular Dystrophy (DMD)
Feb 20, 2025 · Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscular weakness and mild intellectual disability.
Duchenne muscular dystrophy | About the Disease | GARD
Duchenne muscular dystrophy (DMD) affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily in males, though in rare cases may affect females. The …
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Understanding Duchenne Muscular Dystrophy (DMD)
Learn about Duchenne muscular dystrophy (DMD), including its signs and symptoms, the stages of disease progression, and the important role of dystrophin.
Effective Date: 08/08/2024 - BCBSM
- Elevidys is an adeno-associated virus vector-based gene therapy indicated in ambulatory and non-ambulatory patients at least 4 years of age with Duchenne muscular dystrophy (DMD) with …
Regenerative Medicine and Gene Therapy in Japan - PMDA
Gene Therapy in Japan Akira Sakurai, PhD Deputy Reviewer Director, Office of Cellular and Tissue-based Products, ... Brand Name Product Type Approval Date Summary of indication JACE Human …
First Clinical Trial Results of Gene Therapy (GNT0004) for …
Apr 23, 2024 · a collaborative group between sponsors of different gene therapy trials for the same disease. 5 patients aged between 6 and 10 have been treated to date with GNT0004, 4 in France …
This label may not be the latest approved by FDA. For current …
Initial U.S. Approval: 2020 -----INDICATIONS AND USAGE----- VILTEPSO is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients …
Elevidys (delandistrogene moxeparvovec-rokl) (Intravenous)
Nov 13, 2023 · mutation of the DMD gene that is amenable to exon 45 skipping. In much the same way as the exon-skipping therapies, delandistrogene moxeparvovec, the first gene therapy …
Delandistrogene moxeparvovec-rokl (Elevidys) Benefit Criteria
On June 22, 2023, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular …
Elevidys Gene Therapy - Boston Children's Hospital
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Our STN: BL 125781/34 SUPPLEMENT APPROVAL PMR …
Jun 20, 2024 · U.S. Food & Drug Administration 10903 New Hampshire Avenue Silver Spring, MD 20993 www.fda.gov Our STN: BL 125781/34 SUPPLEMENT . APPROVAL PMR FULFILLED
PHARMACY POLICY STATEMENT - CareSource
Jan 1, 2025 · and non-ambulatory pediatric patients at least 4 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. It is the first recombinant gene …
Delandistrogene Moxeparvovec Gene Therapy in Individuals …
This first-in-class gene therapy gained initial accelerated approval by the US Food and Drug Administration (FDA) on June 22, 2023, in 4 –5-year-old am- ... eparvovec, studies reporting …
Gene Therapy Report - Caremark
No Gene therapy, in vivo The treatment of Duchenne muscular dystrophy with a confirmed mutation in the DMD gene Injection-IV, one-time 8,200 adult and pediatric patients 2Q Breyanzi …
Clinical Development of Gene Therapy Products - U.S. Food …
Clinical Development of Gene Therapy Products. 2. ... DMD: Intravenous: Neurotoxicity (Dorsal root ganglia [DRG] ... • Approval of gene therapy products must be based on substantial evidence
CP.PHAR.593 Delandistrogene moxeparvovec-rokl (Elevidys)
Delandistrogene moxeparvovec-rokl (Elevidys) is an adeno-associated virus vector-based gene therapy. FDA Approved Indication(s) Elevidys is indicated in individuals at least 4 years of age: ...
Evaluation of the Gene Therapy Patient Referral Pathways in …
The advancement of gene therapy clinical trials in Duchenne muscular dystrophy (DMD), and the recent accelerated approval of a gene therapy product for use in a limited cohort of DMD …
EXONDYS 51 (eteplirsen) injection, for intravenous use Initial …
Initial U.S. Approval: 2016 ... mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal …
Gene therapy for Duchenne muscular dystrophy
Approval as First Gene Therapy Provide muscles with a healthy copy of the defective gene Gene therapy studies in neuromuscular diseases: SMA1, IBM, BMD, Pompe disease, MTM1 CNM, …
Review - Food and Drug Administration
Jun 25, 2020 · (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Vyondys 53 and Viltepso are approved for the treatment of Duchenne …
Elevidys® (delandistrogene moxeparvovec-rokl)
Jan 4, 2024 · the DMD gene. Elvidys is contraindicated in DMD patients that carry any deletion in exon 8 and/or exon 9 due to serious adverse events of serious liver injury, immune-mediated …
Gene Therapy Report Q1 2025-Q4 2027
Gene Therapy Report Q1 2025-Q4 2027 Projected Treatments and Launch Timelines . 2025 PROJECTED LAUNCHES QUARTER . ... approval 04/29/2025 : New biologic : Yes : Gene therapy, …
An exon-skipping treatment option for DMD patients with an
patients with DMD with an exon 52 deletion6,7 INDICATION VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD …
SPECIALTY GUIDELINE MANAGEMENT
benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy. FDA-Approved Indication Exondys 51 is indicated for the treatment of …
Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, …
an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in …
CP.PHAR.593 Delandistrogene moxeparvovec-rokl (Elevidys)
12. If member is currently on exon skipping therapy (e.g., Amondys 45, Exondys 51, Viltepso, Vyondys 53), member must discontinue therapy prior to Elevidys and not-reinitiate exon skipping …
Duchenne muscular dystrophy gene therapy approval rests …
Duchenne muscular dystrophy gene therapy approval rests on surrogate biomarker data ... DMD gene?” If the committee members vote yes, then SRP-9001 may join the growing list
What is Duchenne Muscular Dystrophy? A DMD Fact Sheet
who have a confirmed mutation of the DMD gene that is amenable to a therapeutic strategy called exon 53 skipping and may help up to 8% of individuals with DMD. Gene Therapy. Elevidys* is the …
022 Gene Therapies for Duchenne Muscular Dystrophy - Blue …
Gene Therapy for DMD The dystrophin gene is one of the largest in the human genome (2.3 million base pairs, over 11,000 of ... Accelerated approval was primarily based on data from Study 1 and …
HIGHLIGHTS OF PRESCRIBING INFORMATION DOSAGE …
Initial U.S. Approval: 2016 . RECENT MAJOR CHANGES . Warnings and Precautions , Hypersensitivity Reactions (5.1) 1/2022 . INDICATIONS AND USAGE EXONDYS 51 is an antisense …
Pharmacy Management Drug Policy - excellusbcbs.com
unmet need for therapy. The accelerated approval of Elevidys was based upon data from two ongoing clinical studies (Study ... Must NOT have a deletion in exon 8 and/or 9 in the DMD gene …
Lethal immunotoxicity in high-dose systemic AAV therapy
AAV gene therapy for many diseases (DMD, SMA, Fabry disease, Danon disease, and methylmalonic acidemia) involving wild-type or engineered AAV capsids. TMA usually occurs within 1–2weeks …
Impact report 2023 - Joining Jack
Funding for gene therapy DMD gene therapy aims to deliver a working version of the dystrophin gene, so that the body can produce functioning dystrophin, which is absent in people with DMD. …
Is dystrophin immunogenicity a barrier to advancing gene …
its approval of Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD), to include both ambulatoryand non-ambulatory individuals aged 4 years and …
RGX-202*, an Investigational Gene Therapy for the Treatment …
RGX-202*, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data. Jahannaz Dastgir, DO. Sr Clinical Development Lead. REGENXBIO. ASGCT, …
Report Overview: Virica's Insights
Sarepta wins “transformational” FDA label expansion for DMD gene therapy Elevidys. The FDA expanded the approval for Elevidys to include all Duchenne Muscular Dystrophy ... • Rocket’s …
Preparing for a future with gene therapy in DMD: A journey …
Preparing for a future with gene therapy in DMD: A journey towards inclusion and equity Monday 28 October, 2024 | 13:00–14:00 AWST Perth Convention and Exhibition Centre, ... Regulatory …
CP.PHAR.593 Delandistrogene moxeparvovec-rokl (Elevidys)
12. If member is currently on exon skipping therapy (e.g., Amondys 45, Exondys 51, Viltepso, Vyondys 53), member must discontinue therapy prior to Elevidys and not-reinitiate exon skipping …
Understanding Gene Therapy - Muscular Dystrophy Association
Q. What is gene therapy? A. ene therapies introduce new genetic G material into a patient’s body to treat or slow the progression of a genetic disease. Q. How does gene therapy work? A.he …
June 22, 2023 Approval Letter - ELEVIDYS
Jun 22, 2023 · dystrophy (DMD) with a confirmed mutation in the DMD gene. The review of this product was associated with the following National Clinical Trial (NCT) numbers: NCT03375164, …
Sarepta Therapeutics Presents Data at the American Society of …
May 16, 2025 · For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD …
Clinical development on the frontier: gene therapy for …
cations per year by 2020, and approval of 10 to 20 cell and gene therapy products by 2025 [29]. This anticipated surge of ... Experience from the development of gene transfer therapy for DMD …
Base editing in humanized dystrophic mice - Cell Press
granted accelerated approval for Sarepta’s Elevidys (AAVrh.74 carrying micro-dystro-phin cDNA) for patients with DMD who are 4–5 years of age.2 This represents an important milestone in DMD …
MVP Health Care Medical Policy Duchenne Muscular …
Approximately 8% of the DMD population have this mutation. Continued FDA approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Viltepso …
Microdystrophin Expression as a Surrogate Endpoint for …
Jan 25, 2023 · microdystrophin gene therapy for DMD. Specifically, we provide support for the use of microdystrophin expression as a surrogate endpoint that could be used in clinical trials to …
The Medical Letter®
gene therapy delandistrogene moxeparvovec-rokl (Elevidys – Sarepta) received accelerated approval from the FDA in 2023 for treatment of ambulatory children 4-5 years old with Duchenne …
Elevidys (delandistrogene moxeparvovec-rokl) - Wellmark
Oct 5, 2023 · Elevidys (delandistrogene moxeparvovec-rokl) is an adenoassociated virus vector- -based gene therapy indicated for: 1. The treatment of ambulatory pediatric patients at least 4 …
Elevidys - modahealth.com
DMD. gene and for the treatment of DMD in patients who are non -ambulatory and have a confirmed mutation in the . DMD . gene. • Delandistrogene moxeparvovec (Elevidys) was initially approved …
Urgent call to action: Making gene therapy for Duchenne …
attention—the exorbitant cost associated with gene therapy for Duchenne muscular dystrophy (DMD). As we celebrate the recent FDA approval of delandistrogene moxeparvovec (Elevidys),1 …
FDA Briefing Document BLA# 125781/00
Corticosteroids are the primary pharmacologic treatment for DMD. Deflazacort (Emflaza) is Food and Drug Administration (FDA)-approved for treatment of DMD in patients 5 years of age and …
Clinical development of novel therapies for Duchenne muscular …
gene therapy, and cell transplantation (Figure 1). The United States ... ceived approval as a novel DMD therapy in Japan and the USA. We are currently developing PMO-based NS-089/NCNP-02, …
This label may not be the latest approved by FDA. For current …
Initial U.S. Approval: 2019 . INDICATIONS AND USAG E. VYONDYS 53 is an antisense oligonucleotide indicated for the treatment of Duchenne muscualr dystrophy (DMD ) in patients …
Urgent call to action: Making gene therapy for Duchenne …
approval of delandistrogene moxeparvovec (Elevidys) (1), the first gene therapy to receive accelerated approval for DMD, it is crucial to recognize the immense financial burden it places on …
