Orphan drug development, rising healthcare costs, patient assistance programs and controversial right-to-try legislation are of paramount interest to the parents and caregivers of boys with Duchenne muscular dystrophy (DMD). ... Read more
Successful results of Translarna (ataluren) and Emflaza (deflazacort) therapies for Duchenne muscular dystrophy (DMD) are highlighted in the 2017 second quarter report by PTC Therapeutics. Orally-administered Translarna is for Duchenne ... Read more
Long after it arrived elsewhere, Emflaza (deflazacort) became the first corticosteroid that the U.S. Food and Drug Administration approved to treat all forms of Duchenne muscular dystrophy — in February 2017. ... Read more
Experts provided an overview of the many approaches to counteract the loss of muscle mass, inflammation, fibrosis, calcium overload, oxidative stress, and ischemia (inadequate blood supply) in Duchenne muscular ... Read more
PTC Therapeutics announced that Emflaza (deflazacort), an approved treatment for all Duchenne muscular dystrophy (DMD) patients regardless of mutation, will be available in a matter of weeks in the U.S., and this time ... Read more
The Duchenne muscular dystrophy (DMD) treatment Emflaza (deflazacort) was successfully acquired by PTC Therapeutics under terms agreed to in March, and may soon become available to patients at, potentially, a new and ... Read more
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 ... Read more
The U.S. Food and Drug Administration (FDA) yesterday announced the approval of the corticosteroid drug deflazacort (under the trade name Emflaza) for the treatment of Duchenne muscular dystrophy (DMD).