Active Ingredient History
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the SMN gene that produces the SMN protein. Wikipedia
Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
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Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
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Spinal Muscular Atrophies of Childhood (approved 2019)
Genetic Therapy (Phase 3)
Muscular Atrophy, Spinal (Phase 4)
Spinal Diseases (Phase 3)
Spinal Muscular Atrophies of Childhood (Phase 4)
Trial | Phase | Start Date | Organizations | Indications |
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