Note: This story was updated May 19, 2025, to clarify the requirements needed for Elevidys to transition to full approval.
Articles by Marisa Wexler, MS
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…
Treatment with DYNE-101 is leading to improvements in muscle function — with notable gains in finger function — among people…
RGX-202, a one-time gene therapy designed to treat Duchenne muscular dystrophy (DMD), has been well tolerated in an ongoing…