FDA approves treatment for Duchenne muscular dystrophy
FDA officials have signed off on eteplirsen, a drug from Sarepta Therapeutics, as an exon-skipping Duchenne therapy. This is the first effective, safe therapy that the FDA has approved for Duchenne in the U.S.
Duchenne, a progressive muscle disorder, causes patients to lose muscle function. This is the most common lethal genetic disorder that people are diagnosed with during childhood. An estimated one out of every 3,500 boys, or 20,000 babies, are born with Duchenne each year around the world.
Eteplirsen has been approved because it can produce dystrophin, which is a muscle-lubricating protein that Duchenne patients lack.
"The FDA approval of a therapy that treats Duchenne is an event I once never imagined I would witness in Jett's lifetime," Christine McSherry, founder of the Jett Foundation and mother of Jett McSherry, who suffers from Duchenne and is participating in a clinical trial for eteplirsen, said. "Not only will eteplirsen's approval change the way every mom and dad reacts when they hear their child's diagnosis; it gives hope to an entire generation of children that they too may have the opportunity to live a fuller and more normal life -- a life where they can be just like their peers for a little while longer. However, the weeks, months and years of unnecessary and burdensome regulatory barriers that eteplirsen faced came at a massive, and unacceptable, human cost to the Duchenne patient community."
Organizations in this story
Jett Foundation 68 Evergreen St Kingston, MA - 02364