"Last year when he won Coach's Choice award, I bawled my eyes out, because his coach said while his body wasn't always able to step up, his ideas, enthusiasm and his mind always do, and he's a real ...

A clinical trial testing Duchenne gene-editing therapy candidate PBGENE-DMD will soon launch in the U.S., having won ...

We were honored to receive the 2025 Clinical Trials Arena R&D Excellence Award in the Precision Endocrine Therapy category, reflecting growing awareness of the utility of (Z)-endoxifen across oncology ...

IMPACT DUCHENNE is a Phase 3 randomized, double-blind, placebo-controlled trial to evaluate the efficacy of a single dose of ...

Columnist Betty Vertin is grateful she's been able to manage cold and flu season at home for her three sons with DMD, and her whole family.

Aucta Pharmaceuticals is pleased to announce the late January launch of PYQUVI oral suspension 22.75 mg/mL.  PYQUVI, a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) ...

Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval SEATTLE, Feb. 5, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc.

Researchers described what they believe to be the first case of SMA type 4 presenting with a false enlargement of a muscle in the calf.

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, announced today that the U.S. Food and Drug Administration (FDA ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...

ELLSWORTH, Maine (WABI) - On Saturday, November 22, those who head to The Grand Theater in Ellsworth will get a chance to see the state of Maine in a way they’ve likely never seen before. “Seasons of ...