Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

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Autores
Categoría Estudio primario
Registro de estudiosclinicaltrials.gov
Año 2018
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). Up to 30 patients \< 6 months (\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.
Epistemonikos ID: 0a958b3a98ce95f05b16b71546aa657cd488d724
First added on: Aug 29, 2023