Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with ...

In September, we shared 24-week dystrophin data from our FORWARD-53 trial, which included highly consistent mean muscle ...

Regulating drug discovery and pharmaceutical manufacturing, the FDA ensures the safety and effectiveness of innovative ...

The US biopharma will discontinue development of the Exondys 51 (eteplirsen) follow up treatment to focus on other treatment ...

Analyst Andrew Fein from H.C. Wainwright maintained a Buy rating on Dyne Therapeutics (DYN – Research Report) and keeping the price ...

The accelerated approval of Exondys 51 is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients. In this case the FDA said Sarepta ...

Sales of Elevidys are quickly gaining ground on Sarepta's exon-skipping trio, which need to be dosed chronically. In the ...

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disorder caused by mutations in a gene resulting in a lack of expressed protein, dystrophin, critical to muscle health. Hereditarily X ...

Furthermore, impressive results in the DMD program, particularly in exon skipping and dystrophin expression ... Its product candidate AOC 1001, is designed to treat a rare monogenic muscle disease.