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Enzyme Therapy In Lysosomal Storage Diseases Proceedings Of The Workshop On Cell Biological And Enzymological Aspects Of The Therapy Of Lysosomal Storage Diseases Leiden The Netherlands April 2 3 1974
Download Enzyme Therapy In Lysosomal Storage Diseases Proceedings Of The Workshop On Cell Biological And Enzymological Aspects Of The Therapy Of Lysosomal Storage Diseases Leiden The Netherlands April 2 3 1974 full books in PDF, epub, and Kindle. Read online Enzyme Therapy In Lysosomal Storage Diseases Proceedings Of The Workshop On Cell Biological And Enzymological Aspects Of The Therapy Of Lysosomal Storage Diseases Leiden The Netherlands April 2 3 1974 ebook anywhere anytime directly on your device. Fast Download speed and no annoying ads. We cannot guarantee that every ebooks is available!
Book Synopsis Enzyme Therapy in Lysosomal Storage Diseases by : Joseph Michel Tager
Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by Joseph Michel Tager and published by . This book was released on 1974 with total page 332 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Enzyme Therapy in Lysosomal Storage Diseases by : J. M. Tager
Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by J. M. Tager and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Enzyme Therapy in Lysosomal Storage Diseases Proceedings of the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases Leiden The Netherlands April 2-3, 1974 by : J. M. Tager
Download or read book Enzyme Therapy in Lysosomal Storage Diseases Proceedings of the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases Leiden The Netherlands April 2-3, 1974 written by J. M. Tager and published by . This book was released on 1974 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:
Author :Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leyden, 1974 Publisher : ISBN 13 :9780444107244 Total Pages :308 pages Book Rating :4.1/5 (72 download)
Book Synopsis Enzyme therapy in lysosomal storage diseases: proceedings for the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leiden by : Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leyden, 1974
Download or read book Enzyme therapy in lysosomal storage diseases: proceedings for the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leiden written by Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leyden, 1974 and published by . This book was released on with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Proceedings of the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leiden, The Netherlands, April 2-3, 1974; and Papers Presented at a Boerhaave Advanced Course on Lysosomal Enzymopathies, Leiden, The Netherlands, April 4-5, 1974 by : W. Th Daems
Download or read book Proceedings of the Workshop on Cell Biological and Enzymological Aspects of the Therapy of Lysosomal Storage Diseases, Leiden, The Netherlands, April 2-3, 1974; and Papers Presented at a Boerhaave Advanced Course on Lysosomal Enzymopathies, Leiden, The Netherlands, April 4-5, 1974 written by W. Th Daems and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Enzyme Therapy in Lysosomal Storage Diseases by : Joseph Michael Tager
Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by Joseph Michael Tager and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis Current Catalog by : National Library of Medicine (U.S.)
Download or read book Current Catalog written by National Library of Medicine (U.S.) and published by . This book was released on with total page 1430 pages. Available in PDF, EPUB and Kindle. Book excerpt: First multi-year cumulation covers six years: 1965-70.
Book Synopsis Enzyme Therapy in Lysosomal Storage Diseases by :
Download or read book Enzyme Therapy in Lysosomal Storage Diseases written by and published by . This book was released on 1974 with total page 308 pages. Available in PDF, EPUB and Kindle. Book excerpt:
Book Synopsis National Library of Medicine Current Catalog by : National Library of Medicine (U.S.)
Download or read book National Library of Medicine Current Catalog written by National Library of Medicine (U.S.) and published by . This book was released on 1971 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: First multi-year cumulation covers six years: 1965-70.
Book Synopsis Lysosomal Storage Disorders by : John A. Barranger
Download or read book Lysosomal Storage Disorders written by John A. Barranger and published by Springer Science & Business Media. This book was released on 2007-10-16 with total page 563 pages. Available in PDF, EPUB and Kindle. Book excerpt: The knowledge of lysosomal biology and the consequences of its dysfunction have increased dramatically in the past 60 years. This book describes the nature of the lysosomal dysfunction and diseases as well as potential future treatments and therapies. Disease specific chapters provide thorough reviews of the clinical features of lysosomal storage disorders, their molecular basis and the commercial or experimental therapeutic approaches sought in this area. This is an invaluable resource for researchers in biochemical and molecular genetics, enzyme therapy, and gene transfer.
Author :U. S. Department of Health and Human Services Publisher :Createspace Independent Pub ISBN 13 :9781483944005 Total Pages :112 pages Book Rating :4.9/5 (44 download)
Book Synopsis Enzyme-Replacement Therapies for Lysosomal Storage Diseases by : U. S. Department of Health and Human Services
Download or read book Enzyme-Replacement Therapies for Lysosomal Storage Diseases written by U. S. Department of Health and Human Services and published by Createspace Independent Pub. This book was released on 2013-03-24 with total page 112 pages. Available in PDF, EPUB and Kindle. Book excerpt: Lysosomes are generally spherical, subcellular organelles bounded by a single layer membrane within eukaryotic cells. They are ubiquitous structures that contain an array of glycoprotein acid hydrolase enzymes, all of which are synthesized in the endoplasmic reticulum and modified in the Golgi apparatus. Lysosomal enzymes catabolize all major classes of biological macromolecules such as proteins, nucleic acids, glycosphingolipids, mucopolysaccharides, and glycogen, as well as sequestered bacteria, viruses, and other foreign substances that are taken up by phagocytosis into white blood cells and macrophages. Lysosomes are also responsible for autophagy, the gradual turnover of each cell's own components as they age and become obsolescent. They may be considered the main site of intracellular digestion and housekeeping. Lysosomal storage diseases (LSDs) comprise a group of unique monogenic autosomal or Xlinked diseases that occur secondary to genetic defects (e.g., single nucleotide substitutions, frameshift mutations, gene deletions) that cause total deficiency or reduced activity of specific native enzymes within the lysosomes. This allows macromolecular compounds that are normally enzymatically catabolized to accumulate within these organelles, expanding them and causing progressive damage in connective tissue, skeletal structure, various organs, and, in some cases, the central nervous system. The damage caused by substrate accumulation results in physical deterioration, functional impairment, and, potentially, death. Some fifty different LSDs have been identified, broadly divided into categories that are defined by accumulation of a specific macromolecule. Although each LSD is individually somewhat rare, as a group they have an incidence of about 1 per 7,000 to 8,000 live births, with regional and genetic population variations. LSDs may be variably expressed as infantile, juvenile, or adult forms. In adult-onset diseases, the pathogenesis is usually slower than in the infantile or juvenile forms, and may include peripheral and CNS symptoms. By contrast, infantile and juvenile forms often involve progressive central nervous system involvement in addition to peripheral symptoms. LSDs also often exhibit significant heterogeneity in ultimate expression, with early or late presentation of symptomatic pathology that may be a function of mutation type and residual enzyme levels. Although specific mutations or types of mutations may be connected to discrete disease effects, genotype-phenotype correlations are often not strong. The objective of this Technical Brief is to provide an overview of FDA-approved ERT for the treatment of six lysosomal storage diseases (shown in Table 1). Four Guiding Questions (following) address the clinical indications for each ERT, potential benefits and harms associated with each ERT product, and dosing and administration details of each ERT. An electronic scan of the literature provides a picture of published evidence on clinical use of these agents for each LSD. This Technical Brief also discusses unresolved or controversial issues surrounding the use of ERT to treat lysosomal storage diseases, based on the literature and information obtained through semi-structured, one-on-one telephone interviews with Key Informants. Guiding Questions include: 1. What FDA-approved enzyme-replacement therapy (ERT) products are available for lysosomal storage diseases (LSDs)? 2. What is the context in which each FDA-approved ERT product is used? 3. What published and unpublished studies have reported on the use and safety of this intervention? 4. What are key unresolved or controversial issues with ERT in LSDs?
Book Synopsis Molecular Basis of Lysosomal Storage Disorders by : John Barranger
Download or read book Molecular Basis of Lysosomal Storage Disorders written by John Barranger and published by Elsevier. This book was released on 2012-12-02 with total page 523 pages. Available in PDF, EPUB and Kindle. Book excerpt: Molecular Basis of Lysosomal Storage Disorders contains the proceedings of the 1983 Conference on the Molecular Basis of Lysosomal Storage Disorders, held at the National Institutes of Health in Bethesda, Maryland. The papers focus on the molecular biology of, and therapeutic approaches to, lysosomal storage disorders, such as mucopolysaccharidoses, sphingolipidoses, and Gaucher disease. Organized into six sections comprised of 29 chapters, this book begins with an overview of enzymes, activator proteins, and stabilizers that underlie lysosomal storage disorders. It then discusses some developments in enzyme purification, receptors for glycoprotein enzymes, factors that control endocytosis, and the intracellular fate of lysosomal hydrolases. Some chapters explain the enzyme biosynthesis, bone marrow transplantation, and enzyme replacement, along with cell hybridization, chromosome localization, phenotype discrimination, and cloning of genes for human lysosomal enzymes. This book is helpful to biochemists, physiologists, pathologists, geneticists, clinical investigators, and practicing physicians concerned with the study, care, and treatment of patients with hereditary metabolic disorders, as well as undergraduate and graduate level students involved in research in this discipline.
Book Synopsis Lysosomal Storage Disorders by : Gregory M. Pastores
Download or read book Lysosomal Storage Disorders written by Gregory M. Pastores and published by World Scientific. This book was released on 2010 with total page 179 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents an overview of lysosomal storage disorders, and provides the reader with an understanding of clinical features, associated complications, and diagnosis and management approaches. It also describes historical developments in the field and current thinking relating to pathophysiology and prospective therapeutic strategies. The book is written by an expert in the field who has been engaged in both basic and clinical research, in addition to having extensive practical experience in patient care. It is written from the perspective of someone who entered the field just as treatment was being introduced, and who has been engaged in the seminal clinical trials and the development of therapeutic guidelines. It offers a broad perspective and should appeal to both novices and experts in the field who seek a single resource that provides a comprehensive picture of relevant topics on this subject. A multi-faceted volume, the author addresses the issue of diagnosis and patient management, underlying mechanisms of disease, sources of morbidity and treatment options, covering issues of interest to both the basic scientist and the clinician. Sample Chapter(s). Foreword (34 KB). Chapter 1: Introduction (1,266 KB). Contents: Clinical Perspectives; Diagnostic Confirmation and Screening Protocols; Assessment of Disease Burden and Assignment of Disease Severity; Pathophysiology and Biomarkers; Current and Emerging Therapies; Future Prospects. Readership: Graduate medical students, nurses, genetic counselors and physicians.
Author :Vladimir P. Torchilin Publisher :Springer Science & Business Media ISBN 13 :3642758215 Total Pages :214 pages Book Rating :4.6/5 (427 download)
Book Synopsis Immobilized Enzymes in Medicine by : Vladimir P. Torchilin
Download or read book Immobilized Enzymes in Medicine written by Vladimir P. Torchilin and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 214 pages. Available in PDF, EPUB and Kindle. Book excerpt: The application of immobilized enzymes in medicine is the main objective of this book. The author reviews natural and synthetic carriers for enzyme immobilization, chemistry of enzyme binding, and in-vitro and in-vivo properties of immobilized enzymes. Four chapters are dedicated to clinical use of immobilized enzymes.
Download or read book National Union Catalog written by and published by . This book was released on with total page 528 pages. Available in PDF, EPUB and Kindle. Book excerpt: Includes entries for maps and atlases.
Book Synopsis Lysosomal Storage Disorders by : Atul B. Mehta
Download or read book Lysosomal Storage Disorders written by Atul B. Mehta and published by John Wiley & Sons. This book was released on 2013-01-22 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Awareness of lysomal storage disorders needs to be raised and there is very substantial pharmaceutical interest to do so. The disorders are often viewed as obscurities but in fact they are treatable. Enzyme replacement therapy is available for four of the disorders and will be available for a further three disorders in the course of the next year. Substrate reduction therapy is licensed for one of them but in the course of the next 12 months it will be licensed for two others and a new form of substrate reduction therapy is being introduced. These diseases present to a very wide range of physicians and paediatricians. Gaucher disease may present to orthopaedic surgeons or haematologists with splenomegaly and/or skeletal disease. However, paediatricians see the childhood variants of Gaucher disease and therefore may present it to neurologists. Fabry disease typically does not present in childhood but presents to adult physicians with end organ damage (renal failrure, cardiac disease, stroke, neuropathy, gastrointestinal symptoms). A text book would draw these divergent strands together. There is substantial scientific interest in these diseases. Gaucher is well recognised as a paradigm of a molecular illness, understood at a basic level which is treatable now with specific therapy and is likely to be treatable with gene therapy within the coming five years. New advances in small molecule therapy – e.g. chaperone treatment, modified antibiotics affecting ribosomal function – are likely to be useful for these diseases in the near future. Trials are already underway. These diseases therefore offer a fabulous platform for teaching modern clinical science from basic genetics right the way through to clinical applications.
Author :Melgardt M. de Villiers Publisher :Springer Science & Business Media ISBN 13 :0387776680 Total Pages :681 pages Book Rating :4.3/5 (877 download)
Book Synopsis Nanotechnology in Drug Delivery by : Melgardt M. de Villiers
Download or read book Nanotechnology in Drug Delivery written by Melgardt M. de Villiers and published by Springer Science & Business Media. This book was released on 2008-10-29 with total page 681 pages. Available in PDF, EPUB and Kindle. Book excerpt: The reader will be introduced to various aspects of the fundamentals of nanotechnology based drug delivery systems and the application of these systems for the delivery of small molecules, proteins, peptides, oligonucleotides and genes. How these systems overcome challenges offered by biological barriers to drug absorption and drug targeting will also be described.