Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for ...
Dystrophin and β-dystroglycan are components of the dystrophin–glycoprotein complex (DGC), a multimolecular assembly that spans the cell membrane and links the actin cytoskeleton to the extracellular ...
Up until now, what was the muscle weakness observed in patients with Duchenne muscular dystrophy thought to be caused by? For twenty years, it has been understood that dystrophin is expressed in ...
PepGen Inc. achieved positive data from the phase 2 CONNECT1 study, using PGN-EDO51 for the treatment of exon 51 skipping DMD patients; higher 10 mg/kg dosing of drug data is expected in early 2025.
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