Overview

This book presents recent advances in translational research on muscular dystrophy (MD) to physicians and researchers, including cutting-edge research on the disease such as regenerative medicine, next-generation DNA sequencing, and nucleic acid therapies. It also describes the current systems for clinical trials and MD patient databases, resources, which will support the early realization of clinical application and improve patients’ quality of life.  MD is the one of the most widely known inherited neuromuscular diseases and is classified into diverse types by symptoms, age of onset, mode of inheritance, and clinical progression. With the development of molecular biology, the occurrence mechanisms of each type of MD are gradually being elucidated. Although there is no known permanent cure yet, the stage of treatment research has now advanced to clinical trials.

Full Product Details

Author:   Shin'ichi Takeda ,  Yuko Miyagoe-Suzuki ,  Madoka Mori-Yoshimura
Publisher:   Springer Verlag, Japan
Imprint:   Springer Verlag, Japan
Edition:   Softcover reprint of the original 1st ed. 2016
Weight:   0.454kg
ISBN:  

9784431566748

ISBN 10:   4431566740
Pages:   199
Publication Date:   31 March 2018
Audience:   Professional and scholarly ,  Professional & Vocational
Format:   Paperback
Publisher's Status:   Active
Availability:   Manufactured on demand  
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Table of Contents

1. Fukuyama congenital muscular dystrophy - Clinical aspects.- 2. α-Dystroglycanopathy.- 3. Myotonic dystrophy.- 4. Molecular pathogenesis and therapeutic strategy in GNE myopathy.- 5. Targeting the type I TGF-β receptor for treating caveolin-3-deficient autosomal dominant limb-girdle muscular dystrophy type 1C and muscle wasting disorders.- 6. Translational Research in Nucleic Acid Therapies for Muscular Dystrophies.- 7. Toward regenerative medicine for muscular dystrophies-Lessons from regeneration processes-.- 8. Stem cell-based therapy for Duchenne muscular dystrophy.- 9. Therapeutic approach of iPS cell technology for treating muscular dystrophy.- 10. Clinical aspects of GNE myopathy and translational medicine.- 11. Patient registries for international harmonized clinical development.- 12. Muscular Dystrophy Clinical Trial Network in Japan.- 13. Translational research on DMD in Japan From mice to exploratory investigator–initiated clinical trial in humans. 

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