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

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 ...

We recently compiled a list of the 10 Most Promising Biotech Stocks According to Hedge Funds. In this article, we are going ...

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 ...

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 ...

Two Chinese biotechs are testing CRISPR gene editing in boys with muscular dystrophy, even as projects at U.S. companies like ...

Peri Finkelstein, 24, has raised more than $1 million to help children with deadly diseases. She shares her inspiring journey with Fox News Digital, detailing what keeps her motivated to "keep ...