Find a Research Study

Find a Research Study

Find a Research Study

Find a Research Study

Find a Research Study

A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED AS AN EARLY INTERVENTION IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY AFTER GENE THERAPY

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.


Why this Research Matters

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.


Who can Participate

All Ages

- 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


Study ID

Protocol Number: 23-2533


Meet the Team

Image of Principal Investigator

Anne Stratton, MD

Principal Investigator