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.
Meet Shamrockstar Henry, whose positive attitude stems from his family’s optimism about potential treatments and cures for Duchenne muscular dystrophy.
Learn why five MDA families are equally dedicated to Muscle Walk — and why you should also support this incredible cause.
PTC Therapeutics announced that the FDA will review its investigational drug ataluren (brand name Translarna) for the treatment of some forms of DMD.
The Labbadia family never wanted to need an MDA Care Center, but when son Brendan was diagnosed with Duchenne muscular dystrophy, they found that their team at Nemours Children’s Hospital in Orlando, Fla., including pediatric neurologist Dr. Richard Finkel, was the one bright spot of an otherwise devastating journey.
Alejandro, who is the local Atlanta-area ambassador for MDA, is one of ten “Shamrockstars” who will be featured on MDA Shamrocks this year at retailers across the country. His gold shamrock will be available exclusively at Lowe’s Home Improvement stores.
Today, Marathon Pharmaceuticals, maker of the recently FDA-approved drug EMFLAZA, announced that they are pausing commercialization efforts in order to address the concerns about price and access to the drug from the Duchenne muscular dystrophy community. MDA will update our community with more information as it becomes available. We remain committed to ensuring that our families have the resources and assistance to begin and maintain treatments with emerging therapies for neuromuscular diseases.
The FDA has approved Marathon Pharmaceuticals’ new drug applications for deflazacort (brand name Emflaza) to treat Duchenne Muscular Dystrophy
The U.S. Food and Drug Administration has granted approval for deflazacort (brand name Emflaza), under development by Marathon Pharmaceuticals, to treat DMD
Raymond (Ray) Walter, who has Duchenne muscular dystrophy, graduated college with degrees in physics, mathematics and economics at age 18. He is currently continuing his doctoral studies in mathematics and physics at the University of Arkansas, where he is a Distinguished Doctoral Fellow and National Science Foundation Graduate Research Fellow. Raymond has also recently been . . .
