Genetech announce new interim data from two studies (JEWELFISH and RAINBOWFISH) of risdiplam (Evrysdi) in spinal muscular atrophy (SMA)

JEWELFISH data show >2-fold increase in survival motor neuron protein levels in a population aged 1-60yrs with SMA who received prior treatment. RAINBOWFISH found pre-symptomatic babies treated with risdiplam for ≥1 year were able to achieve age-appropriate milestones.

SPS commentary:

Risdiplam is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Risdiplam is administered daily at home in liquid form by mouth or by feeding tube.

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