Apply to trial NCT05861986

A few quick questions so the study team can decide if you might be a fit.

RecruitingPhase 4Drug trial

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.

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  2. We forward your profile to the study team

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  3. The team reaches out to schedule screening

    Usually within a few business days, via the contact you give.

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