In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. Becker muscular dystrophy occurs when a ...

Muscular Dystrophy (MD) is more than a medical condition—it's a journey that reshapes lives. Imagine trying to climb stairs, run, or even stand, only to find your muscles not cooperating.

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement and minimal muscle pathology progression.

HOUSTON--(BUSINESS WIRE)--IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new muscle with full length dystrophin in Becker ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX) released topline results from its Phase 2 FIGHT DMD trial. The study evaluated ...

Although the drug increases dystrophin production, which would predict improvement in muscle function, this has not yet been shown. The first gene therapy to be approved for DMD, delandistrogene ...