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Brazilian real-world study of Zolgensma in children with spinal muscular atrophy
RBR-42sf6c5Recruiting
- Sponsor
- Hospital Universitário Professor Edgard Santos
- Start
- Jan 2023
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Countries
- 1
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Overview
Open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), spinal muscular atrophy type 1 and spinal muscular atrophy type 2 (SMA2), sponsored by Hospital Universitário Professor Edgard Santos. Primary endpoints: Real-world effectiveness of onasemnogene abeparvovec, Real-world safety of onasemnogene abeparvovec and 1 more. Started Jan 2023; status: recruiting.
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Drugs
onasemnogene abeparvovec
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Conditions
spinal muscular atrophy (SMA)spinal muscular atrophy type 1spinal muscular atrophy type 2 (SMA2)
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