NBC Montana

Philipsburg family donates pig to local school in late son's memory

Nearly one year ago, Gus Fitz, a Granite High School senior, passed away after a lengthy battle with Duchenne muscular dystrophy.Friends and family describe him ...

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

Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of high unmet clinical need, ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, and Advocacy

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...

Join the First Annual Steps For Hope 5K in Brick, NJ

The inaugural Steps For Hope 5K is set to take place in Brick, New Jersey, organized by LeTip of Brick in partnership with JAR of Hope. This charity run aims to raise awareness and funds for Duchenne ...
Medical Xpress

Mouse model reveals how muscular dystrophy triggers gut muscle over-contraction

Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...
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 ...
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 ...
BioSpace

BridgeBio Pops on Late-Stage Limb-Girdle Muscular Dystrophy Data

New interim data from a Phase III trial puts the company on track to file for FDA approval next year in an indication that not only lacks a disease-modifying treatment but suffered significant ...
STAT

Sarepta to seek approval for gene therapy in rare form of muscular dystrophy

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 ...
Benzinga.com

What's Going On Sarepta Stock On Wednesday?

Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock is trading higher on Wednesday, with apparently no news to justify the movement. This week, Sarepta will present data from its gene therapy, ...
Wane

Form Bio and Cure Rare Disease Partner to Accelerate Genetic Medicine Development, Advancing CRD's Duchenne Muscular Dystrophy Program

Partnership leverages AI-powered genome design to optimize AAV constructs, accelerate lead candidate selection, and improve drug substance manufacturability for rare and ultra-rare neuromuscular ...