The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.

The Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an ...

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

HighTower Advisors LLC decreased its holdings in Sarepta Therapeutics, Inc. (NASDAQ:SRPT – Free Report) by 6.5% during the ...

Sarepta Therapeutics (NASDAQ:SRPT – Get Free Report)‘s stock had its “buy” rating reissued by equities research analysts at ...

After some earlier data sparked questions from analysts, Avidity Biosciences has released more data for its RNA-based ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...

Avidity Biosciences (RNA) announced del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular dystrophy ...

Sarepta shares fell after reporting a patient death linked to Elevidys. The case involved acute liver failure, with a CMV infection as a potential factor.