SMA is a progressive neurodegenerative disease that robs an individual of their ability to walk, eat, and breathe. SMA is the leading genetic cause of death for infants. Symptoms can surface within the first 6 months of life (Type 1, the most severe and common), during the toddler years (Types 2 and 3), or in adulthood (Type 4, the least common form). SMA affects 1 in 11,000 births in the United States each year, and approximately 1 in 50 Americans is a genetic carrier. There are now multiple highly effective treatments for SMA approved by the U.S. Food and Drug Administration (FDA) that make it possible for babies diagnosed with SMA to achieve developmental milestones and individuals with SMA to live full and productive lives.
About Cure SMA
Cure SMA is dedicated to the treatment and cure of SMA. Since 1984, Cure SMA has grown to be the largest network of individuals, families, clinicians, and research scientists working together to advance SMA research, support the full SMA community, and educate public and professional communities about SMA. The organization has directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA, led to breakthroughs in treatment and care, and provides individuals and families the support they need today. For more information, visit https://www.curesma.org/.