The study uncovers the molecular 'rules' driving the arrangement of emerin into nanoclusters and the mechanisms leading to their defective assembly in people with muscular dystrophy. A new ...

Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...

with a new emphasis on Duchenne Muscular Dystrophy. We are excited to have renewed our EU Orphan Drug Designation for rhLAM-111, further validating its potential to transform the lives of those ...

2-4 About Limb-Girdle Muscular Dystrophy Type 2I/R9 LGMD2I/R9 is a rare form of LGMD caused by mutations in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.