MDA Cohosts and Sends Representatives to SMA Patient Focused Drug Development Meeting with FDA

PFDD

Being chosen to speak in front of the FDA and represent the SMA community makes me feel incredibly honored. Today’s SMA Patient-Focused Drug Development (PFDD) meeting with the FDA means we have a voice as patients living with rare muscle diseases. The PFDD meeting was set up so that patients with SMA and the parents of children with SMA could stand before the FDA and voice their concerns, discuss their experiences with research and address the need for continued research that evolves to include more patients. This is my story.

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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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MDA Moves Newborn Screening Forward in Partnership with Cure SMA

This September as we recognize Newborn Screening Awareness month, we are proud to announce a new partnership with Cure SMA aimed at moving newborn screening forward for spinal muscular atrophy (SMA)—the leading genetic cause of death in infants. This is an exciting time for the neuromuscular disease community as decades of research are translating into . . .

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Make Sure Your Voice is Heard. Vote!

Voting is a privilege of living in a democratic society, and, for people with disabilities, it is one of the most important ways to promote leaders that best represent your values. But the voice of the disability community is only as loud and as strong as the number of disability advocates that are registered to . . .

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Developments in SMA Highlight Connections Between Advocacy and Research

Every August is Spinal Muscular Atrophy Awareness Month, and this year we have a lot to celebrate. At the beginning of August, Biogen and IONIS Pharmaceuticals announced encouraging results from a late-stage clinical trial testing an experimental drug for infants with SMA type 1. The drug is called nusinersen and is designed to increase production . . .

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ABLE to Work: Building on the Achieving a Better Life Experience Act of 2014

Data released by the US Department of Labor this month showed that the unemployment rate for people with disabilities is more than double the unemployment rate of those who are considered able bodied. Many factors impact access to employment for those living with disabilities, including a system of benefits and services that are gained and . . .

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