What is Cure SMA?

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that affects the muscles used for activities such as breathing, eating, crawling, and walking. 

Cure SMA is dedicated to the treatment and cure of this devastating disease and provides support for all individuals with SMA and their families. The organization takes a multi-faceted approach to its research, funding basic research, drug
discovery programs, and partnering with leading biotech and pharmaceutical companies to bring new therapies to clinical trials. To date, Cure SMA has
invested more the $82 million in research. 

There’s great reason for hope. Thanks to the dedication of our community and the ingenuity of our researchers, we now have multiple treatments approved by the U.S. Food and Drug Administration that target the underlying genetics of SMA. But our work is not done. Cure SMA is advancing new research that focuses on non-SMN targeted therapies, symptom and muscle-targeting treatments,and the development of combination therapies that increase strength and enable independence for all people with SMA.

Cure SMA also provides those with SMA the practical and tangible support they need to live active, engaged, and hopeful lives. Cure SMA is at the forefront of rare disease advocacy, working directly with legislators, regulators, and officials to advocate on behalf of our community. We also give our community the resources and opportunities to advocate for prompt access to new treatments, adequate funding of critical federal and state programs, and nationwide newborn screening for SMA.


Thank you for your support that has fueled this progress.