Keros Therapeutics Touts Rinvat­ercept DMD Trial Start, Eyes ALS Phase II Talks at Oppenheimer Conference

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 ...

Sarepta Therapeutics to Present New Long-Term and Safety Data Across Gene Therapy and Exon-Skipping Programs at 2026 Muscular Dystrophy Association Clinical & Scientific Congress

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking ...
Charity Today

Stepping up for charity: Stoke fan walking miles ahead of matchday

Trent, is supporting Muscular Dystrophy UK, the leading charity for more than 110,000 children and adults in the UK ...

Woman who faked stage IV breast cancer also 'stole' someone's disease after working at a camp for adults with disabilities

Sarah A. Delashmit was a camp counselor when she struck up a close friendship with Erin Johnson, a woman with cerebral palsy ...
MarketWatch

Atossa Therapeutics Maintains Strong Market Position for (Z)-Endoxifen for Duchenne Muscular Dystrophy as Congress Reauthorizes Priority Review Voucher Program

The MarketWatch News Department was not involved in the creation of this content. Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV ...
Skagit Valley Herald

Atossa Therapeutics Maintains Strong Market Position for (Z)-Endoxifen for Duchenne Muscular Dystrophy as Congress Reauthorizes Priority Review Voucher Program

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.
Morningstar

Cumberland Pharmaceuticals Receives FDA Fast Track Designation for its Ifetroban Duchenne Muscular Dystrophy Program

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 ...
South Bend Tribune

Living with limb girdle muscular dystrophy through hope

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 ...
AZFamily

Arizona State sophomore shares journey as student with muscular dystrophy

TEMPE, AZ (AZFamily) — An Arizona State University student is forging a new path of independence, all while raising awareness for those battling neuromuscular disabilities like himself. On Friday ...
The New York Times

F.D.A. Sharply Limits Use of Drug Linked to Two Teen Deaths

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 ...
The Business Journals

BridgeBio's stock soars after stunning early data against a deadly rare disease

To continue reading this content, please enable JavaScript in your browser settings and refresh this page. Preview this article 1 min The search for a treatment for ...
STAT

BridgeBio study data on drug for rare form of muscular dystrophy could set up approval

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...