"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 ...

PHOENIX — Jace Taylor is a rambunctious 4-year-old boy. He runs, jumps and plays with his friends. He isn’t aware that genetically, he’s different from other children. “I think he’s too young to ...

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 ...

Daughter Verena shares her personal story of living with Limb-girdle type 2A muscular dystrophy. The progressive disease affects muscles around the shoulders, limbs, hips, and thighs, making simple ...