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.

Table of contents (13 chapters)
Fukuyama Congenital Muscular Dystrophy: Clinical Aspects
Ishigaki, Keiko
Pages 1-19
a-Dystroglycanopathy
Kanagawa, Motoi (et al.)
Pages 21-38
Myotonic Dystrophy
Nakamori, Masayuki (et al.)
Pages 39-61
Molecular Pathogenesis and Therapeutic Strategy in GNE Myopathy
Nishimura, Hiroaki (et al.)
Pages 63-75
Targeting the Type I TGF- Receptor for Treating Caveolin-3-Deficient Autosomal Dominant Limb-Girdle Muscular Dystrophy Type 1C and Muscle Wasting Disorders
Ohsawa, Yutaka (et al.)
Pages 77-86
Translational Research in Nucleic Acid Therapies for Muscular Dystrophies
Lee, Joshua J. A. (et al.)
Pages 87-102
Toward Regenerative Medicine for Muscular Dystrophies
Uezumi, Akiyoshi (et al.)
Pages 103-122
Stem Cell-Based Therapy for Duchenne Muscular Dystrophy
Miyagoe-Suzuki, Yuko (et al.)
Pages 123-135
Therapeutic Approach of iPS Cell Technology for Treating Muscular Dystrophy
Sakurai, Hidetoshi
Pages 137-151
Clinical Aspects of GNE Myopathy and Translational Medicine
Mori-Yoshimura, Madoka
Pages 153-163
Patient Registries for International Harmonized Clinical Development
Kimura, En (et al.)
Pages 165-178
Muscular Dystrophy Clinical Trial Network in Japan
Ogata, Katsuhisa
Pages 179-188
Translational Research on DMD in Japan
Takeda, Shin'ichi (et al.)
Pages 189-199