FDA Will Review Translarna for DMD
PTC Therapeutics announced that the FDA will review its investigational drug ataluren (brand name Translarna) for the treatment of some forms of DMD.
Category: News
Keeping Up the Momentum
For this latest round of awards, MDA reviewed 187 applications and had sufficient funds to approve funding for approximately 15 percent of them — 29 grants with a total funding commitment of more than $7 million.
MDA Outlines Principles of Coverage for ACA Replacement with Coalition of Patient Groups
As policy makers are considering various options to modify or replace the Affordable Care Act (ACA), MDA is working to ensure that individuals and families affected by chronic, serious and life-threatening diseases maintain health care access and coverage.
Celebrating Rare Disease Day
MDA families represent more than 40 different rare diseases—as all of the disorders under MDA’s umbrella are classified as “rare.”
Marathon Pharmaceuticals Pauses Commercialization of EMFLAZA to Address Concerns of Duchenne Community
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.
FDA: Emflaza is Approved to Treat DMD in the United States
The FDA has approved Marathon Pharmaceuticals’ new drug applications for deflazacort (brand name Emflaza) to treat Duchenne Muscular Dystrophy
FDA Approves Emflaza for Treatment of 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
A New Resource for MDA Families Living with CMT
Written by and for the CMT community, the new book collects the experiences and advice of those living with CMT on subjects ranging from bracing and stretching to employment and transportation.
Oh, Hello 2017 — I’m Ready for You
When I was asked to continue in my role as National Ambassador for MDA in 2017, I had to think about it for about a nanosecond before I gave my answer. Yes, of course! So it’s official: I’ve re-upped for another tour. 2016 will be a hard act to follow, but now that I’m a registered frequent flyer and have a year of ambassadorship under my belt, I can’t wait to try to top it. In fact, I’ve taken the liberty of compiling a list of experiences I’m looking forward to in 2017. Join me, won’t you?
Spinraza a Breakthrough for SMA
On Dec. 23, 2016 the FDA approved Spinraza for the treatment of SMA. In clinical trials to test Spinraza, participants who received treatment with the drug experienced life-changing outcomes they wouldn’t have been expected to achieve.
