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

In its complete response letter, the FDA cited insufficient evidence establish deramiocel's effectiveness for cardiomyopathy associated with Duchenne muscular dystrophy. The decision comes after CBER ...

TAS-205 showed no significant impact on motor function in patients with Duchenne muscular dystrophy (DMD), highlighting the ongoing search for effective treatments for the rare condition.

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

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli ...

ICER’s upcoming report could shed light on how the launch price of newly approved drugs affects overall cost and access from ...

Capricor Therapeutics (CAPR) on Friday said the U.S. Food and Drug Administration (FDA) has refused to approve its lead cell ...

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the ...

The U.S. Food and Drug Administration has declined to approve Capricor Therapeutics' cell therapy for a heart condition and ...

The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...

The U.S. Food and Drug Administration has declined to approve Capricor Therapeutics' cell therapy for a heart condition, the ...