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

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Autores Novartis
Categoría Estudio primario
Registro de estudiosclinicaltrials.gov
Año 2018
Enlaces Registro de estudios

Este artículo no está incluido en ninguna revisión sistemática

Este artículo es parte de los siguientes hilos de publicación
  • STR1VE-EU [Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion] (3 documentos)
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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