Approved for spinal muscular atrophy (SMA), the efficacy of Evrysdi® in presymptomatic forms remained uncertain. In the international RAINBOWFISH open-label trial, 26 genetically diagnosed infants received Evrysdi® for two years within the first six weeks of life before the onset of symptoms.
- After 12 months of daily oral treatment, 81% of infants could sit unaided for 30 seconds, 54% could stand unaided, and 42% could walk unaided.
- The 23 infants who completed 24 months of treatment did not require permanent respiratory or nutritional support.
- Nine non-serious adverse events related to treatment were reported in seven infants.
Larger controlled studies with longer follow-up will be necessary.
On Clinicaltrials.gov: NCT03779334
