SMA is a motor neurodisease that affects the voluntary muscles that are used for activities such as crawling, walking, head and neck control, and swallowing. Approximately 1 in 6,000 babies are born affected and about 1 in 40 people are genetic carriers.
There are currently no approved treatments and no cure for SMA, and it is the number 1 genetic cause of death in infants under 2 years of age.
Families of SMA funds and directs the leading SMA research programs to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding over half of all the ongoing novel drug programs for SMA.
Mission: Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by:
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