Phase 3 EPIDYS MRI data quantify givinostat’s potential to preserve functional muscle tissue and reduce fat infiltration in patients with ...

NovMetaPharma Co., Ltd. (“NovMetaPharma” or “NMP”), a clinical-stage biopharmaceutical company that develops first-in-class drugs with differentiated mechanisms for metabolic disease, today announced ...

When Angelina Olivera cried out to God and asked why she had been called to help a family member walk the path of Duchenne ...

The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

Gabriel Vergara had always been a starter. His youth baseball career was promising, until his coaches took greater notice of him, but for the wrong reasons. Something was off. An otherwise athletic 11 ...

Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a ...

When my son Hyuk-jun was rushed to the emergency room with a high fever, the attending physician said there was a high ...

Under the agreement, Servier will pay $1.55 billion upfront, with up to an additional $1.1 billion tied to regulatory and commercial milestones.

Company's Biologics License Application on track with PDUFA target action date of August 22, 2026– SAN DIEGO, June 26, 2026 ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which ...

Muscular dystrophy (MD) is a group of genetic diseases that cause your muscles to progressively weaken and degenerate. There are several types of MD, each with its own symptoms, but they all involve ...