Shaping the Future Together

Now, more than ever, we stand ready to shape a future that will change lives, and give new hope to all those who are affected by SMA. Please consider giving generously to help shape our future, together.

113% of $750,000.00 Goal

$853,729.67 Raised

Progress: 114%
Progress: 114%
Raised: $ 853729     Goal: $ 750000

Thanks to the dedication of our families, the generosity of our supporters, and the ingenuity of our research partners, we have reached a new phase in our mission to develop a treatment and cure for SMA.

On December 23, the FDA announced that it has approved Spinraza for SMA, making it the first-ever approved therapy for SMA. This is an incredible milestone for our community.

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.

Shaping the Future of Treatment

Because of the progress in our drug pipeline, we’ve launched a number of urgent initiatives to give drug programs the best chance of success. As part of our patient-focused drug development initiative, we’re working with the FDA to ensure our community’s voice is taken into account when treatments come forward for approval. We’re advocating for these drugs to receive the broadest label possible. And we’re at the forefront of insurance issues, so cost is not a barrier to potentially life-change therapies, ensuring the broadest access to treatment possible.

An FDA-approved treatment will change the course of SMA, but that first approval is not the end of our path. SMA is a complex disease, affecting many different systems, pathways, and processes in the body. To treat all ages, stages, and types of SMA, we’ll need new treatments that address these other areas, working in combination with therapies that address the underlying genetics of SMA.

Shaping the Future of Care

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.

We’re currently working to develop a network of clinical care centers that will collaboratively collect patient data to answer questions and develop strategies for optimal clinical management of SMA. With this project, we hope to bring improved care for SMA to as many families as possible.

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.

Thank You!

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