Muscular Dystrophy Research

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Duchenne Muscular Dystrophy

Author : Camilla Bernardini
Publisher : Humana Press
ISBN 13 : 9781493973736
Total Pages : 287 pages
Book Rating : 4.9/5 (737 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Camilla Bernardini

Download or read book Duchenne Muscular Dystrophy written by Camilla Bernardini and published by Humana Press. This book was released on 2017-10-25 with total page 287 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume explores experimental approaches used to study Duchenne muscular dystrophy (DMD), an X-linked degenerative skeletal muscle disease caused by mutations in the dystrophin gene. Including the latest progress and scientific achievements, the book covers recent discoveries achieved through in vivo gene editing which have proven to be promising in restoring dystrophin expression, at least in ameliorating skeletal muscle symptoms, and the contents focus on “Omics” techniques in gene expression, protein expression, miRNAs, and long non-coding RNA analysis, as well as experimental studies of the structural/functional changes affecting the skeletal and cardiac muscles and ongoing preclinical studies and clinical trials. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Duchenne Muscular Dystrophy: Methods and Protocols serves as a guide for researchers exploring the complicated nature of dystrophin in the hope of helping the victims of this disorder.

Muscular Dystrophy

Author : National Institute of Neurological Diseases and Stroke
Publisher :
ISBN 13 :
Total Pages : 26 pages
Book Rating : 4.:/5 (319 download)

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Book Synopsis Muscular Dystrophy by : National Institute of Neurological Diseases and Stroke

Download or read book Muscular Dystrophy written by National Institute of Neurological Diseases and Stroke and published by . This book was released on 1971 with total page 26 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Muscular Dystrophy

Author : Raymond A. Huml
Publisher : Springer
ISBN 13 : 3319173626
Total Pages : 206 pages
Book Rating : 4.3/5 (191 download)

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Book Synopsis Muscular Dystrophy by : Raymond A. Huml

Download or read book Muscular Dystrophy written by Raymond A. Huml and published by Springer. This book was released on 2015-07-16 with total page 206 pages. Available in PDF, EPUB and Kindle. Book excerpt: This practical and concise guide offers an overview of muscular dystrophy's complicated features, treatment options and general resources. New treatments and a greater understanding of proteins and structures associated with MD are discussed along with long term patient care. Also included are clinical and developmental challenges within the current regulatory landscape and recent scientific and clinical advances. Muscular Dystrophy offers clinicians, researchers, pharmaceutical executives and patient advocacy groups an easy-to-read reference that provides the necessary perspectives of the care giver and patient.

Muscle Gene Therapy

Author : Dongsheng Duan
Publisher : Springer Science & Business Media
ISBN 13 : 144191207X
Total Pages : 281 pages
Book Rating : 4.4/5 (419 download)

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Book Synopsis Muscle Gene Therapy by : Dongsheng Duan

Download or read book Muscle Gene Therapy written by Dongsheng Duan and published by Springer Science & Business Media. This book was released on 2009-11-26 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.

Neuromuscular Disorders of Infancy, Childhood, and Adolescence

Author : Basil T. Darras
Publisher : Elsevier
ISBN 13 : 0124171273
Total Pages : 1156 pages
Book Rating : 4.1/5 (241 download)

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Book Synopsis Neuromuscular Disorders of Infancy, Childhood, and Adolescence by : Basil T. Darras

Download or read book Neuromuscular Disorders of Infancy, Childhood, and Adolescence written by Basil T. Darras and published by Elsevier. This book was released on 2014-12-03 with total page 1156 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neuromuscular disorders are diagnosed across the lifespan and create many challenges especially with infants, children and adolescents. This new edition of the definitive reference, edited by the established world renowned authorities on the science, diagnosis and treatment of neuromuscular disorders in childhood is a timely and needed resource for all clinicians and researchers studying neuromuscular disorders, especially in childhood. The Second Edition is completely revised to remain current with advances in the field and to insure this remains the standard reference for clinical neurologists and clinical research neurologists. The Second Edition retains comprehensive coverage while shortening the total chapter count to be an even more manageable and effective reference. - Carefully revised new edition of the classic reference on neuromuscular disorders in infancy, childhood and adolescence. - Definitive coverage of the basic science of neuromuscular disease and the latest diagnosis and treatment best practices. - Includes coverage of clinical phenomenology, electrophysiology, histopathology, molecular genetics and protein chemistry

Rare Disease Drug Development

Author : Raymond A. Huml
Publisher : Springer Nature
ISBN 13 : 3030786056
Total Pages : 418 pages
Book Rating : 4.0/5 (37 download)

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Book Synopsis Rare Disease Drug Development by : Raymond A. Huml

Download or read book Rare Disease Drug Development written by Raymond A. Huml and published by Springer Nature. This book was released on 2021-11-08 with total page 418 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Duchenne Muscular Dystrophy

Author : Samuel Alexandre Almeida Honorio
Publisher :
ISBN 13 : 9781634821537
Total Pages : 200 pages
Book Rating : 4.8/5 (215 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Samuel Alexandre Almeida Honorio

Download or read book Duchenne Muscular Dystrophy written by Samuel Alexandre Almeida Honorio and published by . This book was released on 2015-04-01 with total page 200 pages. Available in PDF, EPUB and Kindle. Book excerpt: A child with Duchenne Muscular Dystrophy (DMD) faces a childhood and adolescence with a disability that develops gradually. This book intends to expose the problems of children with DMD. The authors discuss situations related to the disease in an attempt to provide some quality and improvement of life for those affected since DMD is a degenerative disease which affects muscle. This book offers a historical study of muscular dystrophy. It also shows some profiles of excellent clinicians and scientists that have contributed to this book with a description of muscular dystrophy from the clinical picture, to the latest techniques in genetics. Also included in the chapters are resolutions of several approaches to DMD, like scales, in order to anticipate how to manage the disease. This book relates some techniques of moderate exercise, like Yoga and water exercises, that are compliable with some advantages in mobility. The book concludes with some current trends and what can be done in the future. This book can be a good read to everyone that is interested in DMD, and also for the ones who know or provide care for those with this terrible disease.

A Guide to Duchenne Muscular Dystrophy

Author : Janet Hoskin
Publisher : Jessica Kingsley Publishers
ISBN 13 : 1784504343
Total Pages : 234 pages
Book Rating : 4.7/5 (845 download)

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Book Synopsis A Guide to Duchenne Muscular Dystrophy by : Janet Hoskin

Download or read book A Guide to Duchenne Muscular Dystrophy written by Janet Hoskin and published by Jessica Kingsley Publishers. This book was released on 2017-12-14 with total page 234 pages. Available in PDF, EPUB and Kindle. Book excerpt: The prognosis for individuals with Duchenne Muscular Dystrophy (DMD) is improving, with some men with DMD living into their 30s and 40s. More vital than ever, this book helps teachers and parents to support children and young people with DMD with their education and transition into adulthood. Leading experts on DMD explain Duchenne and its impact in easy-to-understand terms. Going beyond physical management, particular focus is put on learning and behavioural issues, including speech delay and difficulty learning to read, as well as common comorbid conditions, such as ADHD, autism and OCD. Raising aspirations, the book gives guidance on effective support in the classroom and advice on the transition to adulthood, employment and independent living.

Genetic Neuromuscular Disorders

Author : Corrado Angelini
Publisher : Springer
ISBN 13 : 3319564544
Total Pages : 392 pages
Book Rating : 4.3/5 (195 download)

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Book Synopsis Genetic Neuromuscular Disorders by : Corrado Angelini

Download or read book Genetic Neuromuscular Disorders written by Corrado Angelini and published by Springer. This book was released on 2017-10-05 with total page 392 pages. Available in PDF, EPUB and Kindle. Book excerpt: This updated and expanded new edition of a successful book describes genetic diagnostic entities of neuromuscular disorders. Neuromuscular syndromes are presented clinically either as a case study or as an overview from the literature, accompanied by text presenting molecular defects, and differential diagnosis. This collection of neuromuscular disorders features the differential clinical phenotypes related to each genotype and are representative of the whole spectrum of a genetic muscle disorder, helping the clinician and neuromuscular physician to make a diagnosis. Key points for each genetic disease are identified to suggest treatment, when available, or the main clinical exams useful in follow-up of patients. Genetic Neuromuscular Disorders: A Case-Based Approach is aimed at neuromuscular physicians and neurology residents.

Genome Editing in Neurosciences

Author : Rudolf Jaenisch
Publisher :
ISBN 13 : 9781013268564
Total Pages : 128 pages
Book Rating : 4.2/5 (685 download)

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Book Synopsis Genome Editing in Neurosciences by : Rudolf Jaenisch

Download or read book Genome Editing in Neurosciences written by Rudolf Jaenisch and published by . This book was released on 2020-10-08 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt: Innovations in molecular biology are allowing neuroscientists to study the brain with unprecedented resolution, from the level of single molecules to integrated gene circuits. Chief among these innovations is the CRISPR-Cas genome editing technology, which has the precision and scalability to tackle the complexity of the brain. This Colloque Médecine et Recherche has brought together experts from around the world that are applying genome editing to address important challenges in neuroscience, including basic biology in model organisms that has the power to reveal systems-level insight into how the nervous system develops and functions as well as research focused on understanding and treating human neurological disorders. This work was published by Saint Philip Street Press pursuant to a Creative Commons license permitting commercial use. All rights not granted by the work's license are retained by the author or authors.

Ferri's Clinical Advisor 2020 E-Book

Author : Fred F. Ferri
Publisher : Elsevier Health Sciences
ISBN 13 : 0323679773
Total Pages : 7615 pages
Book Rating : 4.3/5 (236 download)

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Book Synopsis Ferri's Clinical Advisor 2020 E-Book by : Fred F. Ferri

Download or read book Ferri's Clinical Advisor 2020 E-Book written by Fred F. Ferri and published by Elsevier Health Sciences. This book was released on 2019-06-01 with total page 7615 pages. Available in PDF, EPUB and Kindle. Book excerpt: Significantly updated with the latest developments in diagnosis and treatment recommendations, Ferri's Clinical Advisor 2020 features the popular "5 books in 1" format to organize vast amounts of information in a clinically relevant, user-friendly manner. This efficient, intuitive format provides quick access to answers on 1,000 common medical conditions, including diseases and disorders, differential diagnoses, and laboratory tests – all reviewed by experts in key clinical fields. Updated algorithms, along with hundreds of new figures, tables, and boxes, ensure that you stay current with today's medical practice. - Contains significant updates throughout, covering all aspects of current diagnosis and treatment. - Features 27 all-new topics including chronic traumatic encephalopathy, medical marijuana, acute respiratory failure, gallbladder carcinoma, shift work disorder, radial tunnel syndrome, fertility preservation in women, fallopian tube cancer, primary chest wall cancer, large-bowel obstruction, inguinal hernia, and bundle branch block, among others. - Includes a new appendix covering Physician Quality Reporting System (PQRS) Measures. - Provides current ICD-10 insurance billing codes to help expedite insurance reimbursements. - Patient Teaching Guides for many of the diseases and disorders are included, most available in both English and Spanish versions, which can be downloaded and printed for patients.

Muscular Dystrophies—Advances in Research and Treatment: 2012 Edition

Author :
Publisher : ScholarlyEditions
ISBN 13 : 1481623109
Total Pages : 34 pages
Book Rating : 4.4/5 (816 download)

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Book Synopsis Muscular Dystrophies—Advances in Research and Treatment: 2012 Edition by :

Download or read book Muscular Dystrophies—Advances in Research and Treatment: 2012 Edition written by and published by ScholarlyEditions. This book was released on 2012-12-26 with total page 34 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscular Dystrophies—Advances in Research and Treatment: 2012 Edition is a ScholarlyPaper™ that delivers timely, authoritative, and intensively focused information about Muscular Dystrophies in a compact format. The editors have built Muscular Dystrophies—Advances in Research and Treatment: 2012 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Muscular Dystrophies in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Muscular Dystrophies—Advances in Research and Treatment: 2012 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309672988
Total Pages : 127 pages
Book Rating : 4.3/5 (96 download)

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Book Synopsis Exploring Novel Clinical Trial Designs for Gene-Based Therapies by : National Academies of Sciences, Engineering, and Medicine

Download or read book Exploring Novel Clinical Trial Designs for Gene-Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Muscular Dystrophy

Author : Corrado Angelini
Publisher : Nova Science Publishers
ISBN 13 : 9781626184602
Total Pages : 0 pages
Book Rating : 4.1/5 (846 download)

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Book Synopsis Muscular Dystrophy by : Corrado Angelini

Download or read book Muscular Dystrophy written by Corrado Angelini and published by Nova Science Publishers. This book was released on 2013 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Muscular dystrophies include a heterogeneous series of diseases that range from childhood to adult onset cases, of difficult diagnosis and treatment. This book presents the state of the art in muscular dystrophies, after the molecular revolution.

Duchenne Muscular Dystrophy

Author : Alan E. H. Emery
Publisher : OUP Oxford
ISBN 13 : 0191503665
Total Pages : 300 pages
Book Rating : 4.1/5 (915 download)

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Book Synopsis Duchenne Muscular Dystrophy by : Alan E. H. Emery

Download or read book Duchenne Muscular Dystrophy written by Alan E. H. Emery and published by OUP Oxford. This book was released on 2015-02-19 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: Duchenne Muscular Dystrophy, an inherited and progressive muscle wasting disease, is one of the most common single gene disorders found in the developed world. In this fourth edition of the classic monograph on the topic, Alan Emery and Francesco Muntoni are joined by Rosaline Quinlivan, Consultant in Neuromuscular Disorders, to provide a thorough update on all aspects of the disorder. Recent understanding of the nature of the genetic defect responsible for Duchenne Muscular Dystrophy and isolation of the protein dystrophin has led to the development of new theories for the disease's pathogenesis. This new edition incorporates these advances from the field of molecular biology, and describes the resultant opportunities for screening, prenatal diagnosis, genetic counselling and from recent pioneering work with anti-sense oligonucleotides, the possibility of effective RNA therapy. Although there is still no cure for the disorder, there have been significant developments concerning the gene basis, publication of standards of care guidelines, and improvements in management leading to significantly longer survival, particularly with cardio-pulmonary care. The authors also investigate other forms of pharmacological, cellular and gene therapies. Duchenne Muscular Dystrophy will be essential reading not only for scientists and clinicians, but will also appeal to therapists and other professionals involved in the care of patients with muscular dystrophy.

Creatine and Creatine Kinase in Health and Disease

Author : Gajja S. Salomons
Publisher : Springer Science & Business Media
ISBN 13 : 1402064861
Total Pages : 359 pages
Book Rating : 4.4/5 (2 download)

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Book Synopsis Creatine and Creatine Kinase in Health and Disease by : Gajja S. Salomons

Download or read book Creatine and Creatine Kinase in Health and Disease written by Gajja S. Salomons and published by Springer Science & Business Media. This book was released on 2007-10-16 with total page 359 pages. Available in PDF, EPUB and Kindle. Book excerpt: This volume places emphasis on the intricate interplay between creatine and creatine kinase function on one hand and proper brain function, neurodegenerative disease and/or neuroprotection on the other. The book, compiled by outstanding experts, provides a key reference summarizing the state-of-the-art in creatine and creatine kinase research. It is a must-read for understanding the links between creatine metabolism and neuroprotection as well as neurodegenerative disease.

Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001

Author : United States. Congress. House. Committee on Energy and Commerce
Publisher :
ISBN 13 :
Total Pages : 16 pages
Book Rating : 4.:/5 (327 download)

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Book Synopsis Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 by : United States. Congress. House. Committee on Energy and Commerce

Download or read book Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 written by United States. Congress. House. Committee on Energy and Commerce and published by . This book was released on 2001 with total page 16 pages. Available in PDF, EPUB and Kindle. Book excerpt: