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 ...

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Sarepta Therapeutics (NASDAQ:SRPT – Free Report) had its target price lowered by Scotiabank from $105.00 to $80.00 in a ...

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

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

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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT – Get Free Report)’s stock price traded down 4% during trading on Thursday after ...

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

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...