Help Us Shape the Future Together, and Reach our Goal of $750,000

This has been an historic year for the SMA community. On October 28, our pharmaceutical partners at Biogen and Ionis announced that their New Drug Application for Spinraza (nusinersen) was accepted by the FDA. This means we are just one step away from the first FDA-approved treatment for SMA.

What’s more, five other drugs are currently in clinical trials, and one of those, AveXis’s gene therapy program, has received breakthrough designation from the FDA: another first for SMA.

The progress of our drug pipeline has largely been driven by the dedication and generosity of you, our families and supporters. And now, we stand ready to shape a future that will change lives, and give new hope to all those who are affected by SMA.

Your year-end gift will allow us to seize this critical moment. Please consider giving generously to help shape our future, together. 

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We’re working with the FDA to ensure our community’s voice is taken into account when these therapies come forward for approval. We’re advocating for these drugs to receive the broadest label possible. We’re at the forefront of insurance issues, ensuring cost is not a barrier to potentially life-changing therapies. And we’re seeding new basic research to ensure we have treatments that work in combination with those already in the pipeline, for all ages, stages, and types of SMA.

As FDA-approved treatments extend the lives of those with SMA, care will become more and more important. Our goal is not only to extend life, but also to ensure the best possible quality of life. So we’re working to develop a network of clinical care centers to bring improved care for SMA to as many families as possible.

Many of these initiatives, so critical to our community’s goals, did not exist just a few years ago. It speaks to how far we’ve come in our quest to develop a treatment and cure for SMA.

That’s why we’re aiming to raise $750,000 by December 31: so we can build this next phase of treatment and care programs in order to shape a bright future for all those affected by SMA.

For More Information

For more information on how Cure SMA is working shape the future of treatment and care for our community, please see these recent updates from our news section.

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