The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

After hitting a wall with the FDA last year, Novo Nordisk has picked up backing from European drug reviewers for its novel ...

SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy, ...

Scientists have identified previously unreported genes which appear to play a key role in the muscle aging process. It is ...

ALS is a neurodegenerative, life-threatening disease characterized by progressive muscular paralysis caused by the ...

Benitec Biopharma Inc.'s positive results for BB-301 in OPMD treatment show promise, but potential risks and financial ...

Because sickle cell disease isn’t as progressive a disease as others that have gene therapies available, there isn’t as much ...

In this article, we are going to take a look at where Wave Life Sciences (NASDAQ:WVE) stands against other biggest stocks ...

Elizabeth Barker, an accessibility manager at Khan Academy, shares her insights on how ed-tech developers can make their ...

Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...

Mark J. Christman continues to wait, but time is of the essence. Ongoing research and clinical trials are underway, but the U.S. Food and Drug Administration has not yet approved any treatments to ...

MOBILE, Ala. (WKRG) — Firefighters worldwide have raised money for the Muscular Dystrophy Association for 70 years. Mobile Fire-Rescue Capt. Jimmy Connick joined News 5’s Cherish Lombard on ...