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Spinal muscular atrophy (SMA) is a rare, autosomal recessive genetic disease that causes progressive muscle weakness and atrophy. Approximately 6,000 children and adults in the United States are affected, making SMA one of the most common rare diseases. AMCP conducted a virtual Market Insights program to identify perspectives of AMCP members on current and future therapies for SMA.
Proceedings:
Findings from the AMCP Market Insights Program
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