Author Archives: Grace Frank

Viltolarsen Earns European Orphan Drug Designation as DMD Treatment

The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, Nippon Shinyaku announced in a press release. The designation is given to medicinal compounds meant to treat rare conditions that are life-threatening or…

DMD Trial of Dystrophin Gene Therapy SGT-001 Put on Clinical Hold by FDA

The U.S. Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial testing SGT-001, a dystrophin gene therapy intended to treat Duchenne muscular dystrophy (DMD), after the first patient dosed was hospitalized with a possibly serious reaction. Solid Biosciences, the therapy’s developer, announced that within days…

Emflaza Release Put on Hold as DMD Community Reacts to $89K Price Tag

Just days after Emflaza (deflazacort) became the first corticosteroid approved in the U.S. to treat Duchenne muscular dystrophy (DMD) regardless of mutation, the drug’s developer, Marathon Pharmaceuticals, announced it is “temporarily pausing” its sale in the U.S. to address concerns among the DMD community, particularly about the therapy’s high list price…