White Paper:

Spinal Muscular Atrophy: A Rare Disease with a Complex Treatment Landscape (Part 1)

This paper provides an overview of the rare disease, spinal muscular atrophy (SMA).  It describes SMA’s underlying genetic causes, outlines how the three marketed therapeutics work in unique ways to address SMA, and identifies some remaining unmet needs.

Part 2 can be accessed here.  It provides a future outlook on the advancements that could affect the therapeutic and commercial landscape.

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