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.
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.
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?
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.
Strength, Science, and Stories of Inspiration (SSSI) and the Muscular Dystrophy Association (MDA) announced a new research funding mechanism for graduate students and postdoctoral trainees working in the muscle disease field.
MDA is excited to announce that we are accepting applications for the inaugural National Community Advisory Committee, a group that will advise MDA on matters of importance to families living with neuromuscular diseases and help inform MDA’s efforts to support individuals to live unlimited.