This study will evaluate the effectiveness and safety of risdiplam (Evrysdi®) administered to patients under 2 years of age genetically diagnosed with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec.
This is a Phase IV, open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam (Evrysdi®) administered as an early intervention in pediatric patients with SMA who previously received onasemnogene abeparvovec.
- Male and female patients < 2 years of age at the time of informed consent - Genetically confirmed diagnosis of SMA - s received onasemnogene abeparvovec for SMA no less than 3 months, but not more than 7 months, prior to enrollment - Has, in the opini - Provision of Informed Consent Form by a legally authorized representative - Male and female patients < 2 years of age at the time of informed consent - Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous del
Protocol Number: 23-2533
Principal Investigator