Edasalonexent has been shown to preserve muscle function and substantially slow DMD disease progression across all four assessments of muscle function (the North Star Ambulatory Assessment, time to ...

DMD is a rare and incurable pediatric disease caused by mutations in the gene encoding dystrophin, a protein critical for muscle function, including the heart. Patients with DMD slowly lose muscle ...

CAMBRIDGE, England--(BUSINESS WIRE)--bit.bio, the company coding human cells for novel cures, today launches its first set of muscle cell disease model products designed to advance the discovery and ...

NASHVILLE, Tenn., Feb. 4, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...

Summit Therapeutics plcSMMT announced that it has received Rare Pediatric Disease designation from the FDA for one of its pipeline candidates, ezutromid (utrophin modulator), for the treatment of ...

DMD is predominately caused by out-of-frame deletions in the dystrophin gene, which results in absent or defective dystrophin protein, leading to progressive and irreversible muscle function loss. The ...

DURHAM, N.C., June 25, 2025--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene ...

In a world striving for advancement and inclusion, Duchenne muscular dystrophy (DMD) remains a formidable challenge with severe emotional and financial burdens on families and patients. As communities ...