Alex Harold was diagnosed with Duchenne muscular dystrophy at age four and has long been raising funds for Muscular Dystrophy ...

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer ...

Scientists identified a previously unknown gateway into human cells, a receptor called AAVR2, that gene therapy viruses use ...

Del-zota is designed for patients with mutations amenable to exon 44 skipping, which is just 7% of the overall DMD patient ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene ...

From sarcopenia to ALS, researchers are exploring creatine’s broader clinical potential — but more trials are needed to confirm its value.

Scientists in Australia have identified a previously unknown gateway into human cells that could dramatically improve the ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders ...

Insmed Incorporated (NASDAQ:INSM) is one of the stocks on Jim Cramer’s radar. A caller noted that Cramer has not been quick ...

Elaine Chen covers biotech, co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud.

A well-placed biotech investor says U.S. policies are straining life sciences startups — and more disruption is coming.