This article provides key insights and suggested best practices for managed care to improve patient care in MPS disorders, which may also be relevant to other rare and ultra-rare conditions.
Spinal Muscular Atrophy Resources
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.
AMCP Market Insights Summit: Spinal Muscular Atrophy (SMA)
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Proceedings:
Findings from the AMCP Market Insights Program
Related
As part of a multi-year RWE initiative, AMCP Research Institute partnered with IQVIA, payers, pharmaceutical companies, and others to design payer-focused RWE standards that were published in JMCP in September, 2025.
AMCP’s guidance translates CMS’s expectations for patient centered, evidence informed formulary decisions into practical steps payers can act on today without adding unnecessary burden to P&T workflows.
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