Trump's FDA is breaking promises to kids like mine | Opinion

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

Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD and Announces Duchenne Muscular Dystrophy Investor Event

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 ...

Dyne Therapeutics Announces New Positive Cardiopulmonary Results from DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD)

New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history - - Data expand on previously reported results demonstrating that ...
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 ...
WABI-TV

Maine filmmaker living with muscular dystrophy to premiere nature film, proceeds benefiting research

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 ...
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 ...