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 ...

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 ...

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.

Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

EBR Systems is among ASX healthcare stocks with upcoming catalysts, expecting feedback on a FDA substantive review expected ...

Per management, Avidity was the first company to successfully deliver siRNA to skeletal muscles. Preclinical studies showed .

Nashville, Tennessee Friday, November 8, 2024, 13:00 Hrs [IST] ...

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics ...