Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...

Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...

Muscular dystrophy is a disease caused by a genetic mutation. The muscles of muscular dystrophy patients weaken.. | Genetics ...

Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has ...

Physical therapy can help maintain muscle ... MedlinePlus. DMD gene. MedlinePlus. Duchenne muscular dystrophy. National Human Genome Research Institute. About Duchenne muscular dystrophy.

Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD) gene therapy after it failed a phase 3 trial. Fordadistrogene movaparvovec (PF-06939926) was unable to ...