As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

Basel: Roche has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency ...

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

Back in April we first introduced you to Orchard Park teenager Tommy Parzymieso, who the Buffalo Bills selected to announce their first-round pick in this year’s NFL Draft.

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

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

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...

Becker muscular dystrophy is rare. Only about 11,000 people in the U.S. have either BMD or Duchenne muscular dystrophy. Sources Update History. Share. Print ...