Translational Research in Muscular Dystrophy

1. Fukuyama Congenital Muscular Dystrophy: Clinical Aspects
Keiko Ishigaki

2. α-Dystroglycanopathy
Motoi Kanagawa, Tatsushi Toda

3. Myotonic Dystrophy
Masayuki Nakamori, Masanori P. Takahashi

4. Molecular Pathogenesis and Therapeutic Strategy in GNE Myopathy
Hiroaki Nishimura, Satoru Noguchi

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
Yutaka Ohsawa, Shin-ichiro Nishimatsu, Masahiro Fujino, Yoshihide Sunada

6. Translational Research in Nucleic Acid Therapies for Muscular Dystrophies
Joshua J. A. Lee, Toshifumi Yokota

7. Toward Regenerative Medicine for Muscular Dystrophies
Akiyoshi Uezumi, So-ichiro Fukada

8. Stem Cell-Based Therapy for Duchenne Muscular Dystrophy
Yuko Miyagoe-Suzuki, Shin’ichi Takeda

9. Therapeutic Approach of iPS Cell Technology for Treating Muscular Dystrophy
Hidetoshi Sakurai

10. Clinical Aspects of GNE Myopathy and Translational Medicine
Madoka Mori-Yoshimura

11. Patient Registries for International Harmonized Clinical Development
En Kimura, Harumasa Nakamura

12. Muscular Dystrophy Clinical Trial Network in Japan
Katsuhisa Ogata

13. Translational Research on DMD in Japan
Shin’ichi Takeda, Tetsuya Nagata

Avainsanat: Medicine & Public Health, Neurology, Molecular Medicine, Gene Therapy