Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...
Four patients with LGMD-R5 (gamma-sarcoglycanopathy, formerly LGMD-2C) have received the ATA-200 gene therapy as part of the ongoing Phase 1b/2 trial evaluating the safety, pharmacodynamics, and ...
An El Paso mother traveled to Washington, D.C., this week to advocate for her 14-year-old son, who is living with a rare ...
12-year-old Deklan Locke, who has Duchenne muscular dystrophy, uses a power wheelchair and raises awareness about the disease. Duchenne muscular dystrophy is a rare genetic disorder that causes muscle ...
Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorization for Duvyzat (givinostat, Italfarmaco) to treat Duchenne muscular dystrophy ...
The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
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