Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the…
Articles by Marisa Wexler, MS
The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of…
The investigational oral therapy sevasemten significantly reduced markers of muscle damage in people with Becker muscular dystrophy (BMD)…
The venture philanthropy organization SOLVE FSHD is offering $10 million in prizes for innovators who are working to develop…
GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial…
Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys…
The U.S. Food and Drug Administration (FDA) has given Atamyo Therapeutics the go-ahead to start a Phase 1b clinical…
A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially…
CureDuchenne is partnering with Blizzard Entertainment, maker of the massively multiplayer online role-playing game World of Warcraft, on a fundraiser…
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency — again maintained its…