Donations benefit Cure SMA, a national non-profit organization.
SMA is the number one genetic cause of death for infants, and approximately 1 in every 50 individuals is a genetic carrier for SMA. Our son, Rhys, was diagnosed with the most severe type, Type 1, in August 2013. He was with us for 55 beautiful days.
Now there is great reason for hope. We have the first-ever approved treatment that targets the underlying genetics of SMA. Our work is not done. We know what we need to do to develop and deliver effective therapies. And we’re on the verge of further breakthroughs that will continue to change the course of SMA for everyone affected—from infants to adults—and eventually lead to a cure.
We’ve chosen to partner with Cure SMA because of the breadth and depth of their relationships in the world of SMA. Cure SMA is a national non-profit organization that is very involved in working with pharmaceutical companies to develop new treatments for Spinal Muscular Atrophy and help bring them through FDA approval. They’ve already invested over $62 million in research and have funded over half of the ongoing drug programs.
They also provide vital support for families living with SMA, improving their quality of life today. And every year they host the largest SMA conference in the world, bringing families together with the leading SMA researchers.
Thank you for walking every year with us for Rhys and all those with SMA !