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, ...

Sarepta Therapeutics to Report 3-Year Topline Data from EMBARK Study of ELEVIDYS Gene Therapy in Ambulatory Individuals with Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on ...

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 ...
UPI

FDA tightens Duchenne gene therapy after deaths of two teens

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...
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 ...
Times Union

UAlbany, VCU awarded $6.5 million for muscular dystrophy research center

The RNA Institute is located at the University at Albany's uptown campus in Albany, where the Wellstone Center will also have a presence. ALBANY— The University at Albany and Virginia Commonwealth ...
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 ...
Washington Examiner

New York Medicaid is refusing to help children with muscular dystrophy

If a boy in New York has been convinced to think he’s a girl, the state Medicaid program has got you covered. But if a boy with muscular dystrophy just wants to walk again, he’s in trouble. That’s the ...