The HHS secretary’s plans would create a new Administration for a Healthy America (AHA) subagency and slash the department’s workforce by 25%, including 3,500 full-time positions at the FDA.

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent ...

Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...

Regenxbio is ranked Strong Buy due to low EV, strong partnerships, royalty income, bullish trends, and buyout potential.

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II ...

Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the ...

Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Nature Cell Co. Ltd. won U.S. FDA breakthrough therapy designation of its autologous adipose-derived mesenchymal stem cell therapy, Jointstem, March 20, becoming the first Korean company to earn the ...