In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, ...

Keros Therapeutics, Inc. KROS is pushing its lead asset, rinvatercept, into neuromuscular disease with Duchenne muscular ...

Kids with Duchenne muscular dystrophy and their families deserve the chance to decide their own future.

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...

CEO Jasbir Seehra outlined the company’s development priorities and partnership plans during a presentation at Oppenheimer’s 36th Annual Healthcare Life Sciences conference, highlighting plans to init ...

We all know that it is good to exercise for both physical and psychological health. Many of us have also made a promise to start the new year with regular workouts. But many of us do not manage to ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD). But the company’s efforts to expand its use to a new indication is coming ...

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.

NASHVILLE, Tenn., Feb. 4, 2026 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, announced today ...

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 ...