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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

연구 개요

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.

스폰서: 리드: 로슈
적응증: Muscular Atrophy, Spinal
중재: Risdiplam
목표 등록: 28명
시작일: 2024-08-14
완료(예정): 2029-03-31
등록번호: NCT05861999 · ClinicalTrials.gov ↗

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