A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long…
Articles by Marisa Wexler, MS
Enrollment is now ongoing in a first-in-human trial testing EPI-321, an epigenetic therapy designed to address the root cause of…
Note: This story was updated July 30, 2025, to include data from part two of the EMBARK trial. Sarepta…
Sarepta Therapeutics is voluntarily halting all shipments of Elevidys (delandistrogene moxeparvovec-rokl) in the U.S. after a request from the…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for…
The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the…
A second person with Duchenne muscular dystrophy (DMD) has died of acute liver failure after being treated with the…
The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an…
Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy…
CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new gene therapy for…