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.

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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?

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Spinraza a Breakthrough for SMA

Emflaza

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.

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