FDA expands approval of gene therapy for patients with Duchenne Muscular Dystrophy
Elevidys now approved for both ambulatory and non-ambulatory patients aged four and older
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Browsing Tag
Duchenne muscular dystrophy
Pfizer investigational gene therapy for DMD fails in Phase 3 study
The overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable, with mostly mild to moderate…
Givinostat from Italfarmaco gets FDA rare pediatric disease designation in duchenne muscular…
Announces completed enrollment in EPIDYS Phase 3 trial
US FDA approves Viltepso injection for treatment of Duchenne muscular dystrophy mutation
The approval was granted to NS Pharma
FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy…
It is estimated that about 8 percent of patients with DMD have mutation of the dystrophin gene that is amenable to exon 53…