Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...

Biotech stories typically tread a very fine line between promise and pressure. Sometimes a company that’s been a pioneer in ...

The regulator had asked Sarepta Therapeutics to halt all shipments of its therapy, Ele­vidys, after three patients died from ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: ...

The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...

Sarepta Therapeutics, Inc. faces setbacks with Elevidys, safety issues, and workforce cuts, raising concerns about its ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced a strategic restructuring plan ...

Taiho Pharmaceutical has suffered a blow to its near-term pipeline after a drug candidate for Duchenne muscular dystrophy ...

The grandmother of a four-year-old boy from Co Kilkenny diagnosed with a life-limiting genetic disorder has vowed to keep ...

Duchenne muscular dystrophy (DMD) is a genetic disorder caused by mutations in the DMD gene—located on the X chromosome and representing the largest known human gene with 2.4 million base pairs and 79 ...