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Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Novartis Gene Therapies · 2019
ClinicalTrials.gov · Datasets · License: Public Domain · 2019
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spinal-muscular-atrophy
spinal muscular atrophy type i, spinal muscular atrophy, smn2, onasemnogene abeparvovec-xioi, gene replacement therapy, interventional, phase3
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