Articles

Spinal Muscular Atrophy Drug Risdiplam Proves Success in Infants

March 02, 2021

Patients treated with risdiplam at 12 months demonstrated significant improvement in survival and developmental milestones, with 19 of 21 (90%) infants able to survive without permanent ventilation and 7 (41%) infants able to sit without support for at least 5 seconds.