Alfie, from Newry, County Down, was diagnosed with Duchenne muscular dystrophy (DMD) when he was four years old. His dad Jamie Pentony said the new drug, Givinostat, could slow hi ...
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...
Five oculopharyngeal muscular dystrophy (OPMD) patients have now been treated with the gene therapy candidate BB-301 in a ...
Enrollment of healthy volunteers for a Phase 1 trial testing Duchenne muscular dystrophy (DMD) treatment candidate SAT-3247 ...
With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this ...
Muscular dystrophy is a disease caused by a genetic mutation. The muscles of muscular dystrophy patients weaken.. | Genetics And Genomics ...
Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.
Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
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