Sarepta Therapeutics is voluntarily halting all shipments of Elevidys (delandistrogene moxeparvovec-rokl) in the U.S. after a request from the…
Articles by Marisa Wexler, MS
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…
Note: This story was updated May 19, 2025, to clarify the requirements needed for Elevidys to transition to full approval.
The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell…
It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed…