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| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: Continuous Renal Replacement Therapy John A. Kellum, Rinaldo Bellomo, Claudio Ronco, 2016 Continuous Renal Replacement Therapy provides concise, evidence-based, bedside guidance for the management of critically ill patients with acute renal failure, offering quick reference answers to clinicians' questions about treatments and situations encountered in daily practice. |
| enzyme replacement therapy is used to treat: 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 |
| enzyme replacement therapy is used to treat: Immunopharmacology Manzoor M. Khan, 2008-12-19 During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology. |
| enzyme replacement therapy is used to treat: 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 |
| enzyme replacement therapy is used to treat: Fabry Disease Deborah Elstein, Gheona Altarescu, Michael Beck, 2010-08-02 Fabry disease is an X-linked inborn error of metabolism wherein deficiency of a lysosomal enzyme results in systemic deposition of glycosphingolipids. Storage deposition, and hence pathological disease, occurs preferentially in renal glomerular and tubular epithelial cells, myocardial cells, heart valve fibrocytes, neurons of dorsal root ganglia, and in endothelial smooth muscle cells of blood vessels. Thus, Fabry disease is a multi-system disorder, albeit with considerable phenotypic heterogeneity in onset and in severity; however, it is progressive, exhibits extensive morbidity, and is life-threatening. Within the past two decades, there has been a radical change in the natural course Fabry disease by virtue of the availability of specific enzyme replacement therapy. Moreover, there has been a concerted effort to better understand the underlying pathology and equally to identify patients prior to the onset of irreversible end-organ damage. It is to be hoped that the future for patients with Fabry disease can be viewed with greater, albeit guarded, optimism. This state-of-the-art textbook attempts to bridge the span of pre-clinical studies, clinical finding, and management options in a readable but comprehensive manner for the medical practitioner as well as the interested non-medical reader. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: 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 |
| enzyme replacement therapy is used to treat: Pediatric Allergy: Principles and Practice E-Book Donald Y. M. Leung, Hugh Sampson, Raif Geha, Stanley J. Szefler, 2010-10-13 Pediatric Allergy supplies the comprehensive guidance you need to diagnose, manage, and treat virtually any type of allergy seen in children. Drs. Leung, Sampson, Geha, and Szefler present the new full-color second edition, with coverage of the diagnosis and management of anaphylaxis, the immune mechanisms underlying allergic disease, the latest diagnostic tests, and more. Treat the full range of pediatric allergic and immunologic diseases through clinically focused coverage relevant to both allergists and pediatricians. Understand the care and treatment of pediatric patients thanks to clinical pearls discussing the best approaches. Easily refer to appendices that list common food allergies and autoantibodies in autoimmune diseases. Apply the newest diagnostic tests available—for asthma, upper respiratory allergy, and more—and know their benefits and contraindications. Treat the allergy at its source rather than the resulting reactions through an understanding of the immune mechanisms underlying allergic diseases. Get coverage of new research that affects methods of patient treatment and discusses potential reasons for increased allergies in some individuals. Better manage potential anaphylaxis cases through analysis of contributing facts and progression of allergic disease. Effectively control asthma and monitor its progression using the new step-by-step approach. Eliminate difficulty in prescribing antibiotics thanks to coverage of drug allergies and cross-reactivity. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: The Enzyme Cure Lita Lee, 2013-04-24 The medical industry continues to tell us that conventional medicine is the only way to treat all of our health issues. For too many people, however, these treatments do little more than spend money. But there are alternatives. For decades, the use of natural enzymes has been studied and evaluated. The Enzyme Cure is a comprehensive guide for everyone who wishes to learn more about treating health problems with enzymes. The Enzyme Cure teaches you how to use plant enzymes to help reverse asthma, cancer, diabetes, herpes, kidney stones, menopausal symptoms, weight problems, and dozens of other common disorders. It not only details the enzymes that should be used for each condition, but also guides you in treating many underlying problems through diet and lifestyle changes. If you have ever wanted safe and effective medical alternatives, ever wished that doctors would provide new solutions instead of more prescriptions, The Enzyme Cure is for you. |
| enzyme replacement therapy is used to treat: Cells and Culture Thomas Noll, 2010-07-17 Regeneration of tissue to replace damaged or injured tissue is the goal of t- sue engineering. Biomaterials like polyglycolic acid, collagen and small-intestinal submuscosa provide a temporary scaffold to guide new tissue growth and or- nization. Typically, they need to be biodegradable, showing good cell atta- ment and proliferation and they should possess appropriate mechanical properties (Kim et al. , 2000). Synthetic polymers ful ll most of these requirements but lack cell-adhesion peptides on their surface to enhance cell attachment. Ce- adhesion peptides are present in ECM proteins like collagen and elastin. Thus a synthetic polymer coated with ECM proteins would result in a scaffold that mimics the natural cellular environment with enhanced cell attachment and p- liferation. The new bioactive scaffold will be made by combining a synthetic polymer coated with a layer of recombinant ECM proteins produced by CHO cells. The rst step consists of identifying polymers that give best results in terms of CHO cell attachment and growth. Classical techniques to determine biomass are inappropriate to evaluate 3-D structures. Thus a screening system based on stable GFP expressing CHO cells was used to compare the different scaffolds. Simple uorescent measurement after cell lysis allows determining cell attachment and p- liferation on synthetic polymers. Finally CHO cells producing human recombinant collagen I and elastin were generated. We showed that both proteins are expressed and secreted by CHO DG44 cells. 2 Materials and Methods 2. |
| enzyme replacement therapy is used to treat: Monoclonal Antibody Production National Research Council, Institute for Laboratory Animal Research, Committee on Methods of Producing Monoclonal Antibodies, 1999-05-06 The American Anti-Vivisection Society (AAVS) petitioned the National Institutes of Health (NIH) on April 23, 1997, to prohibit the use of animals in the production of mAb. On September 18, 1997, NIH declined to prohibit the use of mice in mAb production, stating that the ascites method of mAb production is scientifically appropriate for some research projects and cannot be replaced. On March 26, 1998, AAVS submitted a second petition, stating that NIH failed to provide valid scientific reasons for not supporting a proposed ban. The office of the NIH director asked the National Research Council to conduct a study of methods of producing mAb. In response to that request, the Research Council appointed the Committee on Methods of Producing Monoclonal Antibodies, to act on behalf of the Institute for Laboratory Animal Research of the Commission on Life Sciences, to conduct the study. The 11 expert members of the committee had extensive experience in biomedical research, laboratory animal medicine, animal welfare, pain research, and patient advocacy (Appendix B). The committee was asked to determine whether there was a scientific necessity for the mouse ascites method; if so, whether the method caused pain or distress; and, if so, what could be done to minimize the pain or distress. The committee was also asked to comment on available in vitro methods; to suggest what acceptable scientific rationale, if any, there was for using the mouse ascites method; and to identify regulatory requirements for the continued use of the mouse ascites method. The committee held an open data-gathering meeting during which its members summarized data bearing on those questions. A 1-day workshop (Appendix A) was attended by 34 participants, 14 of whom made formal presentations. A second meeting was held to finalize the report. The present report was written on the basis of information in the literature and information presented at the meeting and the workshop. |
| enzyme replacement therapy is used to treat: CNS Regeneration Mark H. Tuszynski, Jeffrey Kordower, 1998-10-27 CNS Regeneration focuses on some of the leading current neurological disease models and methods for promoting central nervous system regeneration. Editors and authors are experts in the field, with experience in basic as well as applied neuroscience. In a comprehensive, logical manner, the book unites important basic science advances in neuroscience with novel medical strategies. - The first comprehensive, authoritative volume on the topic of CNS regeneration - Reviews current therapeutic approaches - Editors and authors are experts in the field - Appeals to those interested in basic science as well as those concerned with its medical application |
| enzyme replacement therapy is used to treat: Gaucher's Disease Lunawati L. Bennett, 2020-07-13 Gaucher's Disease: From Diagnosis to Treatment was designed as an educational resource for all professionals involved in the care of patients with Gaucher disease (GD). Chapter 1 is an introduction about GD. GD is the most common autosomal recessive lysosomal storage disease (LSD) due to the deficiency or absence of the activity of enzyme glucosylceramidase (GCase) or also known as acid- glucosidase (GBA1) or uridine phosphate glucosylceramide synthase (UDP-GLC). Defects in these enzymes cause miss-sorting or loss function of lysosomal proteins leading to accumulation of glucocerebroside (GLC) in the tissue macrophages monocyte. Accumulation of GLC cause enlargement of the spleen, destruction of bone, anemia, thrombocytopenia, and abnormalities of the lungs. GD is classified into three types: type 1 GD (GD1) is a chronic and non-neuronopathic accounting for 95% of GD cases, and types 2 and 3 (GD2, GD3) involves nerve cell destruction causing acute brainstem dysfunction or progressive neuroleptic deterioration, respectively. Chapter 1 review disease classification, epidemiology, pathophysiology, and clinical manifestations of GD.Chapter 2 discusses GD diagnosis and clinical presentations from prenatal, newborn, first year of life, childhood, adolescence, and adulthood patients suspected to have GD. Several biomarkers, tools used to detect abnormal biomarkers such as chitotriosidase, pulmonary and activation-regulated chemokines, and other inflammatory markers are discussed, in addition to methods used to detect these biomarkers. Chapter 3 highlights available enzyme replacement therapy (ERT), clinical trials, dosing, and adverse drug reaction of each ERT, pregnancy information, and effect of ERT on endocrine and metabolic profiles. Chapter 4 discusses available substrate reduction therapy (SRT), clinical trials, dosing, adverse drug reaction, pregnancy information, drug-drug interaction and patients' polymorphism profiles that influence the dosing of SRT. Chapter 5 discusses type 2 and 3 GD which are the neuronopathic variants of the GD, genotype and phenotype of the patients, role of ERT or SRT or gene therapy, chaperone therapy, and hematopoietic stem cell transplantation. Chapter 6 discusses common complication of GD such as bone and hepatocellular abnormalities, increased risk of cancer and Parkinson Disease development. Chapter 7 highlights several novel therapies with their mechanisms of action, details regarding ongoing or completed clinical trials such as newer SRT lucerastat which is on clinical trials for possible use in GD3. At this time, there are no drugs available to treat GD2 or GD3. Promising novel therapy include pharmacology chaperone (PC), antioxidants, and gene therapy. There are 2 PCs currently being tested in clinical trial, ambroxol and arimoclomol. Ambroxol, an over the counter drugs has been used to treat various airway infections, showed promising result to cross blood brain barrier that provide promising options for GD3 treatment. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: Medical and Dental Expenses , 1990 |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: The Blood-brain Barrier William M. Pardridge, 1993 The characterization of the blood-brain barrier (BBB) is undergoing a paradigm shift as the century-old concept of a passive, impermeable barrier that segregates blood and brain interstitial fluid is giving way to the idea that the BBB is a dynamic conduit for the transport between blood and brain of those nutrients, peptides, proteins, or immune cells that have access to certain transport systems localized within the BBB membranes. This volume contains 20 contributed chapters organized in four parts: cell-cell interactions, subcellular organelle function, signal transduction mechanisms, and gene expression. There is also a foreword by the late W.H. Oldendorf (1925-1992) on the teleology of the blood-brain barrier and the survival advantage conferred upon the organism by its presence in the vertebrate brain. Annotation copyright by Book News, Inc., Portland, OR |
| enzyme replacement therapy is used to treat: The Complete Book of Enzyme Therapy Anthony J. Cichoke, 1999 Describes a variety of ailments and medical conditions, and lists and current treatments that feature enzymes, vitamins, and minerals |
| enzyme replacement therapy is used to treat: Enzyme-therapy Max Wolf, Karl Ransberger, 1972 |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: Enzymes as Drugs John S. Holcenberg, Joseph Roberts, 1981 |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: 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 |
| enzyme replacement therapy is used to treat: Bone Dysplasias Jürgen W. Spranger, Paula W. Brill, Christine Hall, Gen Nishimura, Andrea Superti-Furga, Sheila Unger, 2018 The definitive guide to genetic bone disorders, now revised and expanded with glossy photographs and radiographs Brilliantly written and produced and deserves to be on the shelves of all pediatric radiologists. It should also be available to geneticists, counselors, and pediatricians. --Radiology This updated and expanded fourth edition of Bone Dysplasias presents age-related radiographs, photographs and clinical guidelines for more than 250 rare constitutional skeletal diseases. Focusing on diagnostically essential imaging and clinical features, each chapter is supplemented with prognostic and therapeutic information, a guide to differential diagnoses, and a short list of the most relevant publications. Organized in accordance with the most recent International Nosology and Classification of Genetic Skeletal Disorders, this new Bone Dysplasias distills the insights of a small, world-class author team on diagnosis and clinical approaches to this most difficult class of disorders. |
| enzyme replacement therapy is used to treat: Pulmonary Function Testing in Children: Techniques and Standards George Polgar, Promadhat Varuni, 1971 |
| enzyme replacement therapy is used to treat: Expression Systems Michael R. Dyson, Yves Durocher, 2007 Protein expression is an increasingly important tool for research on gene function. What is needed is not just a lab manual providing established methods as well as the latest state-of-the-art protocols, but also clear advice on what expression system to choose when. Expression Systems: Methods Expressuniquely fills this need. It covers expression across a broad range of systems, including the following. *Baculovirus expression vectors *CHO cells *E. coli *HEK293-EBNA1 cells *Lactococcus lactis and other gram positive bacteria *S. cerevisiae *transfected insect cells *Pichia pastoris *mammalian cells using BacMam viruses *lentiviral vectors *wheat germ cell-free system The book takes the reader through how to make an informed choice of appropriate system, taking into account the protein target, the time involved, the ultimate use of the expressed protein, and the laboratory equipment required. It also provides step-by-step methods for each system. In addition, the book describes the optimisation of expression strategies, expression engineering using ribosome display, and how to select protein variants with improved expression. Every chapter discusses the merits and limitations of the approaches available, describes the key techniques in full practical detail, and provides sensible advice for immediate use at the bench. In summary, Expression Systems: Methods Expressis a comprehensive laboratory manual and information resource for researchers at all levels, from postgraduate student to principal investigator. |
| enzyme replacement therapy is used to treat: Birth Defects Original Article Series , 1965 |
| enzyme replacement therapy is used to treat: Pancreatic Disease Paul G. Lankisch, Eugene P. DiMagno, 1999-03-29 This symposium volume summarizes the latest research, research that has greatly advanced our understanding of both the basic mechanisms of pancreatic physiology and pathophysiology and the diagnosis and treatment of the diseases of the exocrine pancreas. By doing so, it serves to give direction to future research. Diseases discussed include acute and chronic pancreatitis, cystic fibrosis, and pancreatic cancer. The volume also reflects a central concern of the symposium: to stimulate young persons embarking on a career in this area. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: Nephrology Secrets Edgar V. Lerma, Allen R. Nissenson, MD, FACP, 2011-06-21 Nephrology Secrets, 3rd Edition, by Drs. Edgar V. Lerma and Allen R. Nissenson, gives you the nephrology answers you need to succeed on your rotations and boards.. Its unique, highly practical question-and-answer format, list of the Top 100 Nephrology Secrets, and user-friendly format make it perfect for quick reference. Get the most return for your study time with the proven Secrets® format -- concise, easy to read, and highly effective. Skim the Top 100 Secrets and Key Points boxes for a fast overview of the secrets you must know for success on the boards and in practice. Enjoy faster, easier review and master the top issues in nephrology with mnemonics, lists, quick-reference tables, and an informal tone that sets this review book apart from the rest. Carry it with you in your lab coat pocket for quick reference or review anytime, anywhere. Handle each clinical situation with confidence with chapters completely updated to reflect the latest information. Find the answers you need faster thanks to a new, more streamlined and problem-based organization. Get the high-yield answers you need to address top nephrology questions |
| enzyme replacement therapy is used to treat: Lysosomal Disorders of the Brain Steven U. Walkley, 2004 Lysosomal storage diseases are inherited metabolic disorders characterized by severe pathology, typically involving the brain. Although individually rare, they collectively represent a significant group of diseases that primarily present in early infancy or childhood. In recent yearsconsiderable progress has been made in understanding the molecular mechanisms that lead to disordered function of the lysosomal system and to lysosomal storage. Unravelling the basis for these diseases is providing unique insight into the normal biology of cells and pointing the way to thedevelopment of therapeutic strategies for their treatment. Lysosomal Disorders of Brain details recent advances in the molecular and cellular pathologies of these diseases and in the development of effective therapies. After an overview of the biology of the endosomal-lysosomal system and the types of diseases resulting from defects in this system, thebook describes in detail the molecular mechanisms of storage, model systems and pathophysiological mechanisms, and finally, new advances toward treatment. With each chapter written by leading experts in their field, this book will be valuable for scientists and clinicians in helping them understandthe role of lysosomes in normal cells and mechanisms underlying these disorders, how they can be diagnosed, and the treatment options that are currently available. |
| enzyme replacement therapy is used to treat: 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. |
| enzyme replacement therapy is used to treat: Gaucher Disease, a Century of Delineation and Research Robert J. Desnick, Shimon Gatt, Gregory A. Grabowski, 1982 |
| enzyme replacement therapy is used to treat: Modulation of Protein Function , 1979 |
Enzyme - Wikipedia
An enzyme (/ ˈ ɛ n z aɪ m /) is a protein that acts as a biological catalyst by accelerating chemical reactions. The molecules upon which enzymes may act are called substrates, and the enzyme …
Enzyme | Definition, Mechanisms, & Nomenclature | Britannica
May 20, 2025 · Enzyme, a catalyst that regulates the rate at which chemical reactions proceed in living organisms without itself being altered in the process. Most critically, enzymes catalyze all …
Enzymes: Function, definition, and examples - Medical News Today
Dec 8, 2023 · Enzymes provide help with facilitating chemical reactions within each cell. Since they are not destroyed during the process, a cell can reuse each enzyme repeatedly. This …
Enzymes: What Are Enzymes, Pancreas, Digestion & Liver Function
May 12, 2021 · Enzymes are proteins that help speed up chemical reactions in our bodies. Enzymes are essential for digestion, liver function and much more. Too much or too little of a …
Enzyme: Definition, Types, Structure, Functions, & Diagram
Nov 11, 2021 · Enzymes are protein macromolecules that are necessary to initiate or speed up the rate of chemical reactions in the bodies of living organisms. The molecules on which …
Enzymes - Definition, Examples, Function - Science Notes and …
Mar 25, 2025 · Enzymes are specialized proteins (and in some cases RNA molecules) that act as catalysts in living organisms. They speed up the chemical reactions required for life by …
Enzyme - National Human Genome Research Institute
3 days ago · An enzyme is a biological catalyst and is almost always a protein. It speeds up the rate of a specific chemical reaction in the cell. The enzyme is not destroyed during the reaction …
Enzymes: Structure, Types, Mechanism, Functions - Microbe Notes
Nov 9, 2023 · An enzyme is a protein biomolecule that acts as a biocatalyst by regulating the rate of various metabolic reactions without itself being altered in the process.
What Are Enzymes? - BYJU'S
“Enzymes can be defined as biological polymers that catalyze biochemical reactions.” The majority of enzymes are proteins with catalytic capabilities crucial to perform different …
What is an enzyme? | Britannica - Encyclopedia Britannica
An enzyme is a substance that acts as a catalyst in living organisms, regulating the rate at which chemical reactions proceed without itself being altered in the process. The biological …
Enzyme - Wikipedia
An enzyme (/ ˈ ɛ n z aɪ m /) is a protein that acts as a biological catalyst by accelerating chemical reactions. The molecules upon which enzymes may act are called substrates, and the enzyme …
Enzyme | Definition, Mechanisms, & Nomenclature | Britannica
May 20, 2025 · Enzyme, a catalyst that regulates the rate at which chemical reactions proceed in living organisms without itself being altered in the process. Most critically, enzymes catalyze all …
Enzymes: Function, definition, and examples - Medical News Today
Dec 8, 2023 · Enzymes provide help with facilitating chemical reactions within each cell. Since they are not destroyed during the process, a cell can reuse each enzyme repeatedly. This article...
Enzymes: What Are Enzymes, Pancreas, Digestion & Liver Function
May 12, 2021 · Enzymes are proteins that help speed up chemical reactions in our bodies. Enzymes are essential for digestion, liver function and much more. Too much or too little of a certain …
Enzyme: Definition, Types, Structure, Functions, & Diagram
Nov 11, 2021 · Enzymes are protein macromolecules that are necessary to initiate or speed up the rate of chemical reactions in the bodies of living organisms. The molecules on which enzymes act …
Enzymes - Definition, Examples, Function - Science Notes and …
Mar 25, 2025 · Enzymes are specialized proteins (and in some cases RNA molecules) that act as catalysts in living organisms. They speed up the chemical reactions required for life by lowering …
Enzyme - National Human Genome Research Institute
3 days ago · An enzyme is a biological catalyst and is almost always a protein. It speeds up the rate of a specific chemical reaction in the cell. The enzyme is not destroyed during the reaction and is …
Enzymes: Structure, Types, Mechanism, Functions - Microbe Notes
Nov 9, 2023 · An enzyme is a protein biomolecule that acts as a biocatalyst by regulating the rate of various metabolic reactions without itself being altered in the process.
What Are Enzymes? - BYJU'S
“Enzymes can be defined as biological polymers that catalyze biochemical reactions.” The majority of enzymes are proteins with catalytic capabilities crucial to perform different processes. …
What is an enzyme? | Britannica - Encyclopedia Britannica
An enzyme is a substance that acts as a catalyst in living organisms, regulating the rate at which chemical reactions proceed without itself being altered in the process. The biological processes …
