Most newborns with SMA able to sit after a year of Evrysdi treatment

Positive results have been presented from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA), aged from birth to six weeks.

These data on Roche’s (ROG: SIX) on-market survival motor neuron 2 splicing modifier were presented at the 28th World Muscle Society Congress.

"Evrysdi is the only non-invasive SMA treatment and can be used within hours of birth, potentially allowing these babies to sit, stand and walk, similar to healthy individuals"The RAINBOWFISH study met its primary endpoint, with 80% of babies sitting without support for at least five seconds after one year of Evrysdi treatment.