Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy…
Articles by Marisa Wexler, MS
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…
The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical…
KER-065, a potential treatment for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases, showed a good safety profile in…
The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the first three people with…
Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen the decline in lung…
Patient advocate Donavon Decker has been given the 2025 MDA Legacy Award for Community Impact in Research, recognizing his…