Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA). SMA is a disease that robs people of strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.
Since 1984, we’ve directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA. We've invested over $58 million in research through a comprehensive research model that includes basic research, drug discovery, clinical trials, and clinical care.
We are currently on the verge of breakthroughs in treatment that will strengthen our children’s bodies, extend life, and lead to a cure. There are now 17 ongoing drug programs in the pipeline, and seven of those are in clinical trials. Cure SMA has provided funding for over half of the programs currently in the pipeline.
We have deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and until we have a cure, we’ll do everything we can to support children and families affected by the disease. This includes hosting the Annual SMA Conference each year. The conference is really two separate events—one for families, and one for researchers—that run in parallel, so that researchers and families can interact with and learn from one another. The conference is the world's largest focused on SMA, and it attracts top scientists and clinicians.
Learn more about how you can help us reach a treatment and cure at www.cureSMA.org.