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| fda approved enzyme replacement therapy: 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. |
| fda approved enzyme replacement therapy: Rare Diseases and Orphan Products Institute of Medicine, Board on Health Sciences Policy, Committee on Accelerating Rare Diseases Research and Orphan Product Development, 2011-04-03 Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development. |
| fda approved enzyme replacement therapy: Complementary and Alternative Medicine in the United States Institute of Medicine, Board on Health Promotion and Disease Prevention, Committee on the Use of Complementary and Alternative Medicine by the American Public, 2005-04-13 Integration of complementary and alternative medicine therapies (CAM) with conventional medicine is occurring in hospitals and physicians offices, health maintenance organizations (HMOs) are covering CAM therapies, insurance coverage for CAM is increasing, and integrative medicine centers and clinics are being established, many with close ties to medical schools and teaching hospitals. In determining what care to provide, the goal should be comprehensive care that uses the best scientific evidence available regarding benefits and harm, encourages a focus on healing, recognizes the importance of compassion and caring, emphasizes the centrality of relationship-based care, encourages patients to share in decision making about therapeutic options, and promotes choices in care that can include complementary therapies where appropriate. Numerous approaches to delivering integrative medicine have evolved. Complementary and Alternative Medicine in the United States identifies an urgent need for health systems research that focuses on identifying the elements of these models, the outcomes of care delivered in these models, and whether these models are cost-effective when compared to conventional practice settings. It outlines areas of research in convention and CAM therapies, ways of integrating these therapies, development of curriculum that provides further education to health professionals, and an amendment of the Dietary Supplement Health and Education Act to improve quality, accurate labeling, research into use of supplements, incentives for privately funded research into their efficacy, and consumer protection against all potential hazards. |
| fda approved enzyme replacement therapy: The Neuronal Ceroid Lipofuscinoses (Batten Disease) Sara Mole, Ruth Williams, Hans Goebel, 2011-03-10 The neuronal ceroid lipofuscinoses are an extremely rare group of inherited neurodegenerative diseases that primarily affect children. Core symptoms of these conditions typically include epilepsy, cognitive decline and visual failure. These diseases are so rare that professionals who come into contact with them need a consultative reference work that enables them to become expert, or identify who to contact for more details. Fully updated and revised, this second edition continues to be the definitive volume on this devastating group of disorders. Written by an international collection of authorities in the field, it provides invaluable advice on their diagnosis, patient care, and new treatments that are available. This new edition of the definitive reference text on the neuronal ceroid lipofuscinoses will prove useful for clinicians, family physicians, research scientists, diagnostic laboratories, families affected by the disease as well as by workers in industry planning translational research. |
| fda approved enzyme replacement therapy: Safety of Biologics Therapy Brian A. Baldo, 2016-08-12 This long overdue title provides a comprehensive, up-to-date, state-of-the art review of approved biologic therapies, with coverage of mechanisms of action, Indications for therapy, immunogenicity and a detailed examination of adverse effects and safety of the many and diverse therapeutic agents presented in a total of 13 chapters. It is predicted that by 2016, biologics will make up half of the world's 20 top-selling drugs and by 2018, biologic medicine sales will account for almost half of the world's 100 biggest selling drugs. Recombinant proteins dominate the growing list of the more than 200 approved biotherapeutic agents with targeted antibodies, fusion proteins and receptors; cytokines; hormones; enzymes; proteins involved in blood-clotting, homeostasis and thrombosis; vaccines; botulinum neurotoxins; and, more recently, biosimilar preparations, comprising the majority of approved biologics. Written with clinicians, other health care professionals, and researchers in mind, Safety of Biologics Therapy examines, in a single volume, the full range of issues surrounding the safety of approved biologic therapies. A good understanding of the risks and safety issues of modern biologics therapy is increasingly being demanded of all those connected with their development, handling, prescribing, administration and subsequent patient management. In addition to being of great value to clinicians in all branches of medicine, and to nurses, pharmacists and researchers, this book will prove invaluable for students taking undergraduate and graduate courses in the above disciplines and in the biomedical sciences. |
| fda approved enzyme replacement therapy: 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. |
| fda approved enzyme replacement therapy: Therapeutic Enzymes: Function and Clinical Implications Nikolaos Labrou, 2019-09-03 Therapeutic enzymes exhibit fascinating features and opportunities, and represent a significant and promising subcategory of modern biopharmaceuticals for the treatment of several severe diseases. Research and drug developments efforts and the advancements in biotechnology over the past twenty years have greatly assisted the introduction of efficient and safe enzyme-based therapies for a range of both rare and common disorders. The introduction and regulatory approval of twenty different recombinant enzymes has enabled effective enzyme-replacement therapy. This volume aims to overview these therapeutic enzymes, focusing in particular on more recently approved enzymes produced by recombinant DNA technology. This volume is composed of four sections. Section 1 provides an overview of the production process and biochemical characterization of therapeutic enzymes, while Section 2 focuses upon the engineering strategies and delivery methods of therapeutic enzymes. Section 3 highlights the clinical applications of approved therapeutic enzymes, including aspects on their structure, indications and mechanisms of action. Together with information on these mechanisms, safety and immunogenicity issues and various adverse events of the recombinant enzymes used for therapy are discussed. Section 4, provides discussion on the prospective and future developments of new therapeutic enzymes. This book is aimed at academics, researchers and students undertaking advanced undergraduate/postgraduate programs in the biopharmaceutical/biotechnology area who wish to gain a comprehensive understanding of enzyme-based therapeutic molecules. |
| fda approved enzyme replacement therapy: Hematopoietic Stem Cell Transplantation in Clinical Practice Jennifer G. Treleaven, A. John Barrett, 2008-09-02 A guide to the practice of stem cell transplantation, its status in the treatment of various disorders and the problems that arise after transplantation, aimed at the whole transplant team. - An up to date guide to best practice in the use of stem cell transplantation, covering current status in the treatment of malignant and non-malignant conditions, practical aspects and problems such as infection and graft versus host disease. - Has a practical, accessible approach with free use of algorithms, list tables. - Aimed at the whole transplant team - this is an interdisciplinary field. - International contributor team with editors in the UK and USA. - Illustrated in colour throughout. |
| fda approved enzyme replacement therapy: Safe and Effective Medicines for Children Institute of Medicine, Board on Health Sciences Policy, Committee on Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), 2012-10-13 The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children. |
| fda approved enzyme replacement therapy: 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. |
| fda approved enzyme replacement therapy: Bio-Nanotechnology Manashi Bagchi, Hiroyoshi Moriyama, Fereidoon Shahidi, 2012-11-26 Bio-nanotechnology is the key functional technology of the 21st century. It is a fusion of biology and nanotechnology based on the principles and chemical pathways of living organisms, and refers to the functional applications of biomolecules in nanotechnology. It encompasses the study, creation, and illumination of the connections between structural molecular biology, nutrition and nanotechnology, since the development of techniques of nanotechnology might be guided by studying the structure and function of the natural nano-molecules found in living cells. Biology offers a window into the most sophisticated collection of functional nanostructures that exists. This book is a comprehensive review of the state of the art in bio-nanotechnology with an emphasis on the diverse applications in food and nutrition sciences, biomedicine, agriculture and other fields. It describes in detail the currently available methods and contains numerous references to the primary literature, making this the perfect “field guide” for scientists who want to explore the fascinating world of bio-nanotechnology. Safety issues regarding these new technologies are examined in detail. The book is divided into nine sections – an introductory section, plus: Nanotechnology in nutrition and medicine Nanotechnology, health and food technology applications Nanotechnology and other versatile applications Nanomaterial manufacturing Applications of microscopy and magnetic resonance in nanotechnology Applications in enhancing bioavailability and controlling pathogens Safety, toxicology and regulatory aspects Future directions of bio-nanotechnology The book will be of interest to a diverse range of readers in industry, research and academia, including biologists, biochemists, food scientists, nutritionists and health professionals. |
| fda approved enzyme replacement therapy: Drug Repurposing Farid A. Badria, 2020-12-02 Drug repurposing or drug repositioning is a new approach to presenting new indications for common commercial and clinically approved existing drugs. For example, chloroquine, an old antimalarial drug, showed promising results for treating COVID-19, interfering with MDR in several types of cancer, and chemosensitizing human leukemic cells.This book focuses on the hypothesis, risk/benefits, and economic impacts of drug repurposing on drug discovery in dermatology, infectious diseases, neurological disorders, cancer, and orphan diseases. It brings together up-to-date research to provide readers with an informative, illustrative, and easy-to-read book useful for students, clinicians, and the pharmaceutical industry. |
| fda approved enzyme replacement therapy: The Metabolic & Molecular Bases of Inherited Disease Charles R. Scriver, 2001 Presents clinical, biochemical, and genetic information concerning those metabolic anomalies grouped under inborn errors of metabolism. |
| fda approved enzyme replacement therapy: Antimicrobial Peptides K. Ajesh, K. Sreejith, 2022-11-23 Antimicrobial Peptides: Challenges and Future Perspectives covers the latest developments about antimicrobial peptides in the scenario of drug resistance. The book is divided into 16 chapters arranged in sequence and preceded by chapters on historical developments and their role as regulatory molecules in innate defense mechanism. Emphasis is given to purification techniques and characterization suitable for interdisciplinary research. Chapters provide an inventory of various antimicrobial peptides, from a diverse array of organisms such as bacteria, fungi, insects, amphibians, plants and mammals. A section on marine ecosystem broadens readers understanding on marine based antimicrobial peptides. Additional sections provide an informative overview on peptides with antiviral properties and those targeting multi-drug resistant bacteria. Recent reports and mechanism on resistance against antimicrobial peptides are also provided, along with key insights into the challenges and future perspectives of peptide drug development. - Emphasizes antimicrobial peptides targeting various human viruses and multidrug resistant bacteria - Written by internationally recognized experts who provide readers with a wide and useful perspective - Provides in-depth resources for undertaking a research work in antimicrobial peptides with the inclusion of chapters on purification techniques and structural details - Addresses the possibility and availability of peptide antibiotics in the global drug market - Serves as a complete resource from the discovery to drug development of peptide antibiotics |
| fda approved enzyme replacement therapy: The Gerson Therapy Charlotte Gerson, Morton Walker, 2001 Offers a nutritional program that utilizes the healing powers of organic fruits and vegetables to reverse the effects of cancer and other illnesses. |
| fda approved enzyme replacement therapy: Bad Bug Book Mark Walderhaug, 2014-01-14 The Bad Bug Book 2nd Edition, released in 2012, provides current information about the major known agents that cause foodborne illness.Each chapter in this book is about a pathogen—a bacterium, virus, or parasite—or a natural toxin that can contaminate food and cause illness. The book contains scientific and technical information about the major pathogens that cause these kinds of illnesses.A separate “consumer box” in each chapter provides non-technical information, in everyday language. The boxes describe plainly what can make you sick and, more important, how to prevent it.The information provided in this handbook is abbreviated and general in nature, and is intended for practical use. It is not intended to be a comprehensive scientific or clinical reference.The Bad Bug Book is published by the Center for Food Safety and Applied Nutrition (CFSAN) of the Food and Drug Administration (FDA), U.S. Department of Health and Human Services. |
| fda approved enzyme replacement therapy: Molecular and Genetic Basis of Renal Disease David B. Mount, Martin R. Pollak, 2008-01-01 This companion to Brenner and Rector's The Kidney offers a state-of-the-art summary of the most recent advances in renal genetics. Molecular and Genetic Basis for Renal Disease provides the nephrologist with a comprehensive look at modern investigative tools in nephrology research today, and reviews the molecular pathophysiology of the nephron as well as the most common genetic and acquired renal diseases. A comprehensive clinical review of Medelian renal disease is also be included. Detailed review of the molecular anatomy and pathophysiology of the nephron that provides relevant basic science to consider when diagnosing and managing patients with these disorders. |
| fda approved enzyme replacement therapy: Drug-Induced Liver Injury , 2019-07-13 Drug-Induced Liver Injury, Volume 85, the newest volume in the Advances in Pharmacology series, presents a variety of chapters from the best authors in the field. Chapters in this new release include Cell death mechanisms in DILI, Mitochondria in DILI, Primary hepatocytes and their cultures for the testing of drug-induced liver injury, MetaHeps an alternate approach to identify IDILI, Autophagy and DILI, Biomarkers and DILI, Regeneration and DILI, Drug-induced liver injury in obesity and nonalcoholic fatty liver disease, Mechanisms of Idiosyncratic Drug-Induced Liver Injury, the Evaluation and Treatment of Acetaminophen Toxicity, and much more. - Includes the authority and expertise of leading contributors in pharmacology - Presents the latest release in the Advances in Pharmacology series |
| fda approved enzyme replacement therapy: Enzymes as Drugs John S. Holcenberg, Joseph Roberts, 1981 |
| fda approved enzyme replacement therapy: Preclinical Safety Evaluation of Biopharmaceuticals Joy A. Cavagnaro, 2013-03-07 The goal is to provide a comprehensive reference book for the preclinicaldiscovery and development scientist whose responsibilities span target identification, lead candidate selection, pharmacokinetics, pharmacology, and toxicology, and for regulatory scientists whose responsibilities include the evaluation of novel therapies. —From the Afterword by Anthony D. Dayan Proper preclinical safety evaluation can improve the predictive value, lessen the time and cost of launching new biopharmaceuticals, and speed potentially lifesaving drugs to market. This guide covers topics ranging from lead candidate selection to establishing proof of concept and toxicity testing to the selection of the first human doses. With chapters contributed by experts in their specific areas, Preclinical Safety Evaluation of Biopharmaceuticals: A Science-Based Approach to Facilitating Clinical Trials: Includes an overview of biopharmaceuticals with information on regulation and methods of production Discusses the principles of ICH S6 and their implementation in the U.S., Europe, and Japan Covers current practices in preclinical development and includes a comparison of safety assessments for small molecules with those for biopharmaceuticals Addresses all aspects of the preclinical evaluation process, including: the selection of relevant species; safety/toxicity endpoints; specific considerations based upon class; and practical considerations in the design, implementation, and analysis of biopharmaceuticals Covers transitioning from preclinical development to clinical trials This is a hands-on, straightforward reference for professionals involved in preclinical drug development, including scientists, toxicologists, project managers, consultants, and regulatory personnel. |
| fda approved enzyme replacement therapy: Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics Reed E. Pyeritz, Bruce R. Korf, Wayne W. Grody, 2024-10-23 For decades, Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics has served as the ultimate resource for clinicians integrating genetics and genomics into medical practice. With detailed coverage in contributions from more than 250 of the world's most trusted authorities in medical genetics and a series of 11 volumes available for individual sale, the Seventh Edition of this classic reference includes the latest information on seminal topics such as prenatal diagnosis, genome sequencing, public health genetics, genetic counseling, and management and treatment strategies to complete its coverage of this growing field for students, health providers, and researchers involved in the care of patients with genetic conditions, and increasingly, all areas of health and disease. This comprehensive yet practical resource emphasizes theory and research fundamentals related to the applications of medical genetics and genomics across the full spectrum of inherited disorders and applications to medicine more broadly. In this volume, leading physicians and researchers thoroughly examine medical genetics and genomics as applied to developmental disorders, as well as genetic conditions that affect hearing and vision. Here genetic researchers, students, and health professionals will find new and fully revised chapters on human developmental genetics, disorders affecting craniofacial development, chromosomal abnormalities, including aneuploidies and structural abnormalities, hereditary hearing impairment, and various genetic conditions of the eye. With regular advances in genomic technologies propelling precision medicine into the clinic, Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics, Seventh Edition bridges the gap between high-level molecular genetics and practical application and serves as an invaluable clinical tool for health professionals and researchers. · Thoroughly introduces genetic researchers, students, and healthcare professionals to the principles of human developmental genetics · Examines a wide range of developmental disorders, including craniofacial development as well as disorders affecting hearing and vision · Includes color images supporting identification, concept illustration, and method processing · Features contributions by leading international researchers and practitioners of medical genetics |
| fda approved enzyme replacement therapy: Transporters as Drug Targets Gerhard F. Ecker, Rasmus P. Clausen, Harald H. Sitte, 2017-04-10 As opposed to other books on the topic, this volume is unique in also covering emerging transporter targets. Following a general introduction to the importance of targeting transporter proteins with drugs, the book systematically presents individual transporter classes and explains their pharmacology and physiology. The text covers all transporter families with known or suspected importance as drug targets, including neurotransmitter transporters, ABC transporters, glucose transporters and organic ion transporters. The final part discusses recent advances in structural studies of transport proteins, assay methods for transport activity, and the systems biology of transporters and their regulation. With its focus on drug development issues, this authoritative overview is required reading for researchers in industry and academia targeting transport proteins for the treatment of disease. |
| fda approved enzyme replacement therapy: Bioprospecting of Enzymes in Industry, Healthcare and Sustainable Environment Hrudayanath Thatoi, Sonali Mohapatra, Swagat Kumar Das, 2021-01-23 The rapid urbanization and industrialization of developing countries across the globe have necessitated for substantial resource utilization and development in the areas of Healthcare, Environment, and Renewable energy. In this context ,this resourceful book serves as a definitive source of information for the recent developments in application of microbial enzymes in various sectors. It covers applications in fermentation processes and their products, extraction and utilisation of enzymes from various sources and their application in health and biomass conversion for production of value added products. Different chapters discuss various areas of bioprospecting in enzyme technology, and describe why these are the mainstays for industrial production of value added products. The rich compilation of the cutting-edge advances and applications of the modern industrial based techniques hold feasible solutions for a range of current issues in enzyme technology. This book will be of particular interest for scientists, academicians, technical resource persons, engineers and members of industry. Undergraduate and graduate students pursuing courses in the area of industrial biotechnology will find the information in the book valuable. General readers having interest towards biofuels, enzyme technology, fermented food and value added products, phytochemicals and phytopharmaceutical products will also find the book appealing. Readers will discover modern concepts of enzymatic bioprocess technology for production of therapeutics and industrial value added products. |
| fda approved enzyme replacement therapy: Landmark Papers in Internal Medicine Harold C. Sox, Edward J. Huth, American College of Physicians (2003- ), 2009 ACPs journal, Annals of Internal Medicine, is one of the most prestigious journals in medicine. This new book looks at the landmark papers published in Annals, as selected by leading experts from each subspecialty of internal medicine, and how they impacted (and continue to influence) medical science. |
| fda approved enzyme replacement therapy: Physician's Guide to the Treatment and Follow-Up of Metabolic Diseases Nenad Blau, Georg F. Hoffmann, J.V. Leonard, Joe T. R. Clarke, 2006-01-16 This reference provides concise information on the treatment and management of inherited metabolic diseases for the clinician. World experts cover all commonalities of therapy giving practical advice and guidance for daily practice. All established treatment protocols in this quickly developing area of medicine are clearly described, including follow-up protocols and monitoring. Alternative and experimental therapies are also described and evaluated. Numerous tables, figures, and several indices (symptom, disease name, tests, etc.) allow rapid access to specific details. This book is invaluable to anyone dealing with patients with inherited metabolic diseases, pediatricians, internists, neurologists, and clinical geneticists. |
| fda approved enzyme replacement therapy: The Nonhuman Primate in Nonclinical Drug Development and Safety Assessment Joerg Bluemel, Sven Korte, Emanuel Schenck, Gerhard Weinbauer, 2015-03-13 The Nonhuman Primate in Drug Development and Safety Assessment is a valuable reference dedicated to compiling the latest research on nonhuman primate models in nonclinical safety assessment, regulatory toxicity testing and translational science. By covering important topics such as study planning and conduct, inter-species genetic drift, pathophysiology, animal welfare legislation, safety assessment of biologics and small molecules, immunotoxicology and much more, this book provides scientific and technical insights to help you safely and successfully use nonhuman primates in pharmaceutical toxicity testing. A comprehensive yet practical guide, this book is intended for new researchers or practicing toxicologists, toxicologic pathologists and pharmaceutical scientists working with nonhuman primates, as well as graduate students preparing for careers in this area. - Covers important topics such as species selection, study design, experimental methodologies, animal welfare and the 3Rs (Replace, Refine and Reduce), social housing, regulatory guidelines, comparative physiology, reproductive biology, genetic polymorphisms and more - Includes practical examples on techniques and methods to guide your daily practice - Offers a companion website with high-quality color illustrations, reference values for safety assessment and additional practical information such as study design considerations, techniques and procedures and dosing and sampling volumes |
| fda approved enzyme replacement therapy: Artificial Cells, Cell Engineering and Therapy S Prakash, 2007-05-31 Artificial cells, cell engineering and therapy are emerging technologies which will make a significant impact on the future of medicine and healthcare. However, research within the field is vast. This unique book provides a comprehensive study of the most recent advances in the field and its practical applications.The first part of the book offers the reader an introduction to the basics of artificial cell technology with chapters on its origins, design, current status within medicine and future prospects. Part two covers apoptosis, the use of bone marrow stromal cells in myocardial regeneration together with signalling and tissue engineering. Part three discusses artificial cells for therapy, procedures for various clinical conditions and the current status of the discipline within the field. The book concludes with a final section on the role of artificial cells in medicine with particular focus on the use of artificial cells as blood substitutes and their potential use in myocardial regeneration, drug delivery and in treating kidney and bowel diseases, diabetes and cancer.Artificial cells, cell engineering and therapy is a valuable reference for researchers, students and practitioners within the field. - Introduces the basics of artificial cell technology - Provides a comprehensive study of the most recent advances in artificial cells, cell engineering and cell therapy - Discusses the design, engineering and uses of artificial cells |
| fda approved enzyme replacement therapy: A Rare Breed Daniel S. Levine, Daniel P. Maher, 2017-08-15 BioMarin Pharmaceutical Inc., named by Forbes magazine as one of the world¿s ten most innovative companies, is a world leader in developing therapies to treat rare orphan genetic diseases. A Rare Breed recounts the first 20 years of its history, the struggles it faced in developing drugs for diseases that represented markets most pharmaceutical companies saw as too small to pursue, and how it grew into a global enterprise valued at more than $16 billion. It follows the researchers, patients, and executives who played a role in taking novel ideas forward and how they overcame scientific, regulatory, and financial challenges to bring breakthrough therapies to children who needed them. |
| fda approved enzyme replacement therapy: Biologics and Biosimilars Xiaodong Feng, Hong-Guang Xie, Ashim Malhotra, Catherine F. Yang, 2022-06-13 Biologics and Biosimilars: Drug Discovery and Clinical Applications is a systematic integration and evaluation of all aspects of biologics and biosimilars, encompassing research and development, clinical use, global regulation, and more. Biosimilars are biological therapeutic agents designed to imitate a reference biologic with high similarities in structure, efficacy, and safety, but also with potential clinical effective and cost-efficient options for the manufacturers, payers, clinicians, and patients. Most of the top-selling prescription drugs in the current market are biologics, which have revolutionized the treatment strategies and modalities for life-threatening and/or rare diseases. This book outlines the key processes and challenges in drug development, regulations, and clinical applications of biologics, biosimilars, and even interchangeable biosimilars. Global experts in the field discuss essential categories and prototype drugs of biologics and biosimilars in clinical practice such as allergenics, blood and blood components, cell treatment, gene therapy, recombinant therapeutic proteins or peptides, tissues, and vaccines. Additional features: Integrates the latest bench and bedside evidence of drug development and regulations of biologics and biosimilars Contains key study questions for each chapter to guide the readers, as well as drug charts for all therapeutic applications of biologics and biosimilars Presents detailed schematic illustrations to explain the drug development, clinical trials, regulations, and clinical applications of biologics and biosimilars This book is an invaluable tool for health care professional students, providers, and pharmaceutical and health care industries, as well as the public, providing readers with educational updates about the drug development and clinical affairs of biological medications and their similar drugs. |
| fda approved enzyme replacement therapy: 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. |
| fda approved enzyme replacement therapy: Vademecum Metabolicum Johannes Zschocke, Georg Friedrich Hoffmann, 2004 Inborn errors of metabolism represent a special challenge in general and paediatric practice. The development and prognosis of the affected child may depend on rapid and effective treatment, but the large number of genetic disorders in various biochemical pathways makes it difficult to be familiar with diagnostic strategies and specific therapies. With this in mind, the Vademecum Metabolicum aims to provide practical guidance to the clinician. The second edition has been extensively updated and expanded. The first section summarises the differential diagnosis and management strategies for a large number of clinical situations, and describes indications and requirements for all relevant metabolic investigations. The second section explains the metabolic pathways in detail. Clinical characteristics, diagnostic approaches and the basis of therapy are discussed for more than 300 individual disorders. Foreword by James V. Leonard, London |
| fda approved enzyme replacement therapy: Mucopolysaccharidoses Update (2 Volume Set) Shunji Tomatsu, Roberto Giugliani, Tadao Orii, Maurizio Scarpa, Paul Harmatz, Christine Lavery, Grzegorz Wegrzyn, Mucopolysaccharidoses (MPS) are caused by a deficiency of lysosomal enzyme activities needed to degrade glycosaminoglycans (GAGs), which are long unbranched polysaccharides consisting of repeating disaccharides. GAGs include: Chondroitin sulfate (CS), dermatan sulfate (DS), heparan sulfate (HS), keratan sulfate (KS), and hyaluronan. Their catabolism may be blocked singly or in combination depending on the specific enzyme deficiency. There are eleven known enzyme deficiencies, resulting in seven distinct forms of MPS with a collective incidence higher than 1 in 25,000 live births. Accumulation of undegraded metabolites in lysosomes gives rise to distinct clinical syndromes. Generally, the clinical conditions progress if untreated, leading to developmental delay, systemic skeletal deformities, and early death.Other clinical features include coarse facial features, corneal clouding, recurrent ear and nose infections, inguinal and umbilical hernias, hepatosplenomegaly, heart valvular disease and skeletal deformities. Clinical features related to bone lesions may include marked short stature, cervical stenosis, pectus carinatum, small lungs, joint rigidity (but laxity for MPS IV), kyphoscoliosis, lumbar gibbus, and genu valgum. Patients with MPS are often wheelchair-bound and physical handicaps increase with age as a result of progressive skeletal dysplasia, abnormal joint mobility, and osteoarthritis. Patients may need multiple orthopedic procedures including cervical decompression and fusion, carpal tunnel release, hip reconstruction and replacement, and femoral or tibial osteotomy throughout their lifetime. Current measures to intervene in bone disease progression and CNS involvement are not perfect and palliative, and improved therapies are urgently required and are being proposed.Enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT), and gene therapy are available or in development for some types of MPS. Delivery of sufficient enzymes to the brain and bones, especially avascular cartilage, to prevent or ameliorate the devastating neurological defects and skeletal dysplasias remains an unmet challenge. The use of an anti-inflammatory drug is also under clinical study. Therapies should start at a very early stage prior to irreversible bone lesion and damage, since the severity of CNS involvement and skeletal dysplasia is associated with the level of activity in a patient's daily life.For the maximum benefit of available therapies, early detection and intervention are critical. Newborn screening and diagnostic systems have been developed by using tandem mass spectrometry. We review the history of diagnosis and newborn screening as well. Overall, this book illustrates a to-date overview of the pathogenesis, diagnosis, biomarkers, screening, and updated therapies as well as their impact on MPS, including ERT, HSCT, gene therapy, and anti-inflammatory drugs. History and activities of MPS societies are also described. It is a comprehensive textbook meant to cover many areas in the field of MPS and appeals to a broad spectrum of readers including physicians, scientists, students, pharmaceutical companies, and MPS communities. |
| fda approved enzyme replacement therapy: Intravenous Lipid Emulsions P.C. Calder, D.L. Waitzberg, B. Koletzko, 2014-11-26 Lipids have been in clinical use as components of intravenous nutrition for over 50 years. Over the last 15 years, new and improved lipids that include olive oil and/or fish oil have replaced the more traditional ones. These new lipids offer the opportunity to deliver high amounts of fatty acids and possess different functional properties: in particular, they can influence inflammatory processes, immune responses and hepatic metabolism. This book brings together articles written by leading international authorities in the area of intravenous lipids. Contributions discuss the latest findings in the field, ranging from pre-clinical research to the most recent clinical trials. Lipid functionality and utility in pediatric, adult surgical and critically ill patients are covered, as is the use of lipids in long-term home parenteral nutrition. Addressing a broad spectrum of topics, this publication provides a wealth of information for basic scientists, clinical researchers and clinical practitioners alike. |
| fda approved enzyme replacement therapy: Pharmacogenomics Yui-Wing Francis Lam, Larisa H. Cavallari, 2013-02-12 Pharmacogenomics: Challenges and Opportunities in Therapeutic Implementation includes discussions and viewpoints from the academic, regulatory, pharmaceutical, clinical, socio-ethical and economic perspectives. Each chapter presents an overview of the potential or opportunity within the areas discussed and also outlines foreseeable challenges and limitations in moving pharmacogenomics into drug development and direct therapeutic applications. This edited book contains review questions for a more in-depth analysis of the implications of pharmacogenomics and discussion points to generate ideas on best to move the field forward. Clinical pearls and case studies are used to illustrate real-life experiences and both successful and unsuccessful applications. Tables, figures, and annotations are included throughout the book to facilitate understanding and further reference. - Multi-contributed book and chapters are written by contributors who are experts in their field - Provides perspectives from those involved in all aspects of pharmacogenomics—including academic, regulatory, economic, industry and medical—to illustrate how all of the pieces fit together and where the challenges may be - Includes case studies of both successful and unsuccessful applications so readers can consider the potential and challenges in moving the science into drug development and direct therapeutic applications - Chapters contain discussion questions and clinical pearls and enable readers to reflect on how to move pharmacogenomics forward and apply these observations and useful tips to their own work and research |
| fda approved enzyme replacement therapy: Pharmaceutical Medicine and Translational Clinical Research Divya Vohora, Gursharan Singh, 2017-11-14 Pharmaceutical Medicine and Translational Clinical Research covers clinical testing of medicines and the translation of pharmaceutical drug research into new medicines, also focusing on the need to understand the safety profile of medicine and the benefit-risk balance. Pharmacoeconomics and the social impact of healthcare on patients and public health are also featured. It is written in a clear and straightforward manner to enable rapid review and assimilation of complex information and contains reader-friendly features.As a greater understanding of these aspects is critical for students in the areas of pharmaceutical medicine, clinical research, pharmacology and pharmacy, as well as professionals working in the pharmaceutical industry, this book is an ideal resource. - Includes detailed coverage of current trends and key topics in pharmaceutical medicine, including biosimilars, biobetters, super generics, and - Provides a comprehensive look at current and important aspects of the science and regulation of drug and biologics discovery |
| fda approved enzyme replacement therapy: Elsevier's 2022 Intravenous Medications - E-Book Shelly Rainforth Collins, 2021-04-16 Find the essential information you need to safely administer more than 400 intravenous drugs! For more than 45 years, Gahart’s 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, the 2022 edition includes approximately 10 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, and with its history of impeccable accuracy, Gahart’s annual publication gives you the extra confidence and guidance you need to safely and effectively treat patients. Monographs on more than 400 IV drugs offer 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 provided on the companion Evolve website. 45-year history of impeccable accuracy reinforces the importance of safe IV drug administration. Perfect depth of information equips you with everything that is needed 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 newly approved drugs by the FDA provide 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. |
| fda approved enzyme replacement therapy: Newborn Screening for Pompe Disease Wuh-Liang Hwu, Yin-Hsiu Chien, Raymond Wang, 2021-09-02 Pompe disease, also known as acid maltase deficiency or acid alpha-glucosidase deficiency, in its most severe form results in a rapidly progressive, neonatal-onset skeletal and cardiomyopathy, leading to early infantile death without treatment. The development of treatment with recombinant enzyme replacement therapy radically transformed the clinical trajectory of those affected, enabling long-term ventilator-free survival with resolution of cardiomyopathy. These positive clinical outcomes resulted in the implementation of newborn screening programs for Pompe disease across the world. This Special Issue highlights some of the experiences of Pompe screening programs worldwide and discusses public policy and ethical issues elicited by presymptomatic screening for Pompe disease. |
| fda approved enzyme replacement therapy: 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. |
| fda approved enzyme replacement therapy: Novel Therapeutic Concepts in Targeting Glioma Faris Farassati, 2012-04-04 Novel Therapeutic Concepts for Targeting Glioma offers a comprehensive collection of current information and the upcoming possibilities for designing new therapies for Glioma by an array of experts ranging from Cell Biologists to Oncologists and Neurosurgeons. A variety of topics cover therapeutic strategies based on Cell Signaling, Gene Therapy, Drug Therapy and Surgical methods providing the reader with a unique opportunity to expand and advance his knowledge of the field. |
| fda approved enzyme replacement therapy: Krause and Mahan's Food and the Nutrition Care Process, 16e, E-Book Janice L Raymond, Kelly Morrow, 2022-07-30 **Selected for Doody's Core Titles® 2024 with Essential Purchase designation in Nutrition** Provide optimal nutritional care with the latest guidelines to evidence-based practice! Krause and Mahan's Food & the Nutrition Care Process, 16th Edition provides an all-in-one resource for the dietetics information you need to care for patients throughout the entire life cycle. With insight from clinical specialists, the book guides you through the steps of assessment, diagnosis and intervention, monitoring, and evaluation. It also covers nutrition in each stage of life, weight management, medical nutrition therapies for conditions and disorders, and the use of nutrition therapies in childhood. From a team of nutrition experts led by Janice L. Raymond and Kelly Morrow, this classic text has been trusted by nurses, nutritionists, and dieticians for since 1952. - UNIQUE! Pathophysiology algorithms and flow charts present the cause, pathophysiology, and medical nutrition management for a variety of disorders and conditions to help you understand illness and provide optimal nutritional care. - Clinical case studies help you translate academic knowledge into practical patient care using a framework of the nutrition care process. - Sample Nutrition Diagnosis boxes present a problem, its etiology, and its signs and symptoms, then conclude with a nutrition diagnosis, providing scenarios you may encounter in practice. - Clinical Insight boxes expand on information in the text, highlight new areas of focus, and contain information on studies and clinical resources. - New Directions boxes suggest areas for further research by spotlighting emerging areas of interest in nutrition care. - Focus On boxes provide thought-provoking information on key nutrition concepts. - Summary boxes highlight CRISPR, the Indigenous food movement, hearing assessment, health disparities, and the Health At Every Size movement, and include a tribute to Dr. George Blackburn, a respected specialist in obesity and nutrition. - Key terms are listed at the beginning of each chapter and bolded within the text. - NEW Infectious Diseases chapter is written by a new author with specific expertise in infectious disease. - NEW Transgender Nutrition chapter is added, from two new authors. - NEW! COVID-19 updates are provided in multiple chapters, each relating to epidemiology and patient care. - NEW! Information on the FODMAP diet is included in the appendix, covering the sugars that may cause intestinal distress. - NEW! Emphasis on diversity, equity, and inclusion is included in all chapters. - NEW! Updated International Dysphagia Diet Standardisation Initiative (IDDSI) information is included in the appendix. - NEW! Updated pregnancy growth charts are added to this edition. - NEW! Updated Healthy People 2030 information is added throughout the book. |
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The FDA approved the first nasal spray for the emergency treatment of allergic reactions, including those that are life-threatening, in adult and pediatric patients who weigh at least 30 …
