Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense (or premature . . .
PTC Therapeutics President and CEO Stuart Peltz, Ph.D., issued a letter to the DMD community after the FDA declined to approve ataluren (brand name Translarna) for the treatment of DMD.
PTC Therapeutics reached an agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza™ (deflazacort), the first treatment approved in the U.S. for all Duchenne muscular dystrophy (DMD) patients five years and older, regardless of their genetic mutation.
PTC Therapeutics announced today that it recently has submitted an appeal to escalate continuing discussions with the U.S. Food and Drug Administration (FDA) about a path toward approval in the United States for Translarna to treat some forms of Duchenne muscular dystrophy (DMD). PTC completed submission of its New Drug Application (NDA) to market Translarna . . .
Background: PTC Therapeutics yesterday announced results from the phase 3 “ACT DMD” trial of ataluren, an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). The trial results showed clinically meaningful benefits for ataluren-treated patients, indicating the drug may slow functional decline in DMD patients, with some patients apparently . . .
