U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s ...

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

Capricor Therapeutics (NASDAQ:CAPR) received the Orphan Drug designation from the U.S. FDA for its lead cell therapy candidate, Deramiocel, to treat becker muscular dystrophy. With its orphan drug ...

The publication of this Infographic was funded by Edgewise Therapeutics. This infographic presents an overview of Becker Muscular Dystrophy outlining the disease itself, exploring the diagnosis of the ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in ...

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...

Sarepta Therapeutics has suspended shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, to nonambulatory patients following a second reported death due to acute liver failure. The ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...