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

Good morning, and welcome to the Wave Life Sciences Third Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen only mode. As a reminder, this call is being ...

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

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 ...

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 ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

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

WALTHAM, Mass., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...

This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication ...

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