Continued improvements in motor milestones reported in infants with Type 1 Spinal Muscular Atrophy after two years of treatment with risdiplam

According to data from the FIREFISH trial, presented at conference, 93% (38/41) of infants were alive and 83% (34/41) alive without permanent ventilation at 24 months of treatment. In addition, 92% maintained the ability to feed orally; further motor milestones were also reached.

Biospace Inc.