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Background and purpose
Spinal muscular atrophy (SMA) is caused by reduced levels of survival of motor neuron (SMN) protein due to deletions and/or mutations in the SMN1 gene. Risdiplam is an orally administered molecule that modifies SMN2 pre‐mRNA splicing to increase functional SMN protein.
Methods
SUNFISH Part 1 was a dose‐finding study conducted in 51 individuals with types 2 and 3 SMA aged...
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