“November 1, 2011 was the day that everything changed for our family. We were sitting in a room with no windows and seemingly no air to breathe and the news came like a bulldozer: Duchenne muscular dystrophy, a muscle wasting disease with no cure that is 100% fatal. I couldn’t believe it. All I wanted to do . . .
The U.S. Food and Drug Administration (FDA) has accepted Marathon Pharmaceuticals’ New Drug Applications for deflazacort for the treatment of Duchenne muscular dystrophy (DMD) and granted Priority Review. Deflazacort, a glucocorticoid, works as an anti-inflammatory and immunosuppressant. In the United States, the drug is considered an investigational therapy, as it has not been approved by . . .
Researchers are looking for boys and men, ages 5 to 18 years, to participate in a natural history study that is designed to assess the potential of imaging techniques to monitor disease progression and serve as an outcome measure for clinical trials in Duchenne muscular dystrophy (DMD). Both healthy volunteers and individuals with DMD are . . .
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 . . .
A passionate ATV rider and supportive big brother, 14-year old Devin Argall is defying the odds at every turn. The Wisconsin State Goodwill Ambassador was diagnosed with Duchenne muscular dystrophy (DMD) at the age of two, and doctors told him he would likely need a wheelchair by 10 years old. Today, Devin continues to walk . . .
The Lybrand family motto is “finish strong,” which, for them, means never giving up and always turning can’ts into cans. It’s no wonder, then, that the youngest Lybrand, 6-year-old Ethan, is a living embodiment of the Live Unlimited spirit. Just three days before his second birthday, Ethan was diagnosed with Duchenne muscular dystrophy. Although the deadly disease will gradually . . .
Earlier this Spring, I had the honor of taking part in the 3rd annual Strength, Science and Stories of Inspiration event at the Harvard Science Center in Cambridge, Massachusetts. As a patient with dysferlinopathy (a form of muscular dystrophy), I was heartened to see every seat in the 500-person auditorium filled to support the mission of finding . . .
Marathon Pharmaceuticals announced today it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of people with Duchenne muscular dystrophy (DMD). The FDA has 60 days to determine whether the NDA is complete and acceptable for filing. Deflazacort, a glucocorticoid, works . . .
Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has requested that the company provide dystrophin data from biopsies that were taken as part of the ongoing confirmatory study of eteplirsen, called PROMOVI. The data was requested by the agency in connection with its ongoing evaluation of Sarepta’s New Drug Application (NDA) . . .
BioMarin Pharmaceutical announced May 31 that it is discontinuing development of its exon skipping drug drisapersen. The company recently withdrew its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting that indicated the CHMP intended to issue a negative . . .
