Treatment with oral therapy ifetroban led to improvements in measures of heart function in people with Duchenne muscular dystrophy…
Articles by Marisa Wexler, MS
The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate for myotonic…
Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the…
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…