February marks Rare Disease Month, a time to amplify the voices of the 300 million people worldwide living with a rare disease—including the individuals in the U.S. impacted by spinal muscular atrophy (SMA). Throughout this month, and especially during Rare Disease Week (starting February 23rd), we’re committed to advancing research, raising awareness, and advocating for policies that improve the lives of those living with SMA.
Research: The Key to Future Treatments
One of the biggest priorities of Rare Disease Month is shining a light on the power of research to drive progress. Thanks to continued investments in SMA research, we've already seen three FDA-approved treatments—a remarkable achievement in the rare disease space. But there’s more work to be done.
Cure SMA continues to fund research that:
- Explores new treatment approaches, including combination therapies.
- Investigates the role of the SMN protein to deepen our understanding of SMA.
- Develops better tools and models to track disease progression and improve patient care.
Your support fuels the next breakthrough, improving the outcomes and quality of life for the SMA community.
Advocacy: Turning Awareness into Action
Beyond research, Rare Disease Month is also about advocacy—ensuring that those impacted by SMA have access to the resources, treatments, and policies they need.
Cure SMA is actively working to:
- Expand access to vital support services for youth and adults with SMA.
- Advance policies that create more accessibility in public transportation, especially air travel.
- Ensure full and timely access to SMA treatments, therapies, and vital equipment.
As we approach Rare Disease Week, we’ll be amplifying these efforts on a national level, engaging policymakers, and advocating for meaningful change.
Your Support Makes Progress Possible
This Rare Disease Month, you can help drive real progress for the SMA community. Whether you donate to fund SMA research, share your story to raise awareness, or stay informed on the latest advancements, every action makes a difference.
💜 Join us in making an impact this Rare Disease Month - together, we will create a better future for everyone living with SMA.