Translational Research in Muscular Dystrophy (2016) Shin’ichi Takeda

Description

Focuses on recent advancements in translational research concerning muscular dystrophies. Topics include the identification of disease-causing genes (like DMD and α-dystroglycanopathies), molecular and cellular mechanisms, and new therapeutic strategies such as exon skipping therapy and stem cell-based therapy. The text also covers international efforts in clinical research and patient registries for intractable neuromuscular diseases.