pharmaphorum

Solid Bio hit as FDA slaps clinical hold on DMD gene therapy

The FDA has placed another clinical hold on a gene therapy for Duchenne muscular dystrophy developed by Solid Biosciences, pulverising its share price. The halt of the IGNITE DMD trial was prompted by ...
pharmaphorum

FDA okays Italfarmaco’s non-steroidal DMD therapy Duvyzat

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD), becoming the first non-steroidal option for patients with all genetic ...