Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
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DOI: 10.3233/jnd-221560
Abstract: Background: Spinal muscular atrophy (SMA) is a neuromuscular disorder arising from biallelic non-functional survival motor neuron 1 (SMN1) genes with variable copies of partially functional SMN2 gene. Intrathecal onasemnogene abeparvovec administration, at fixed, low doses,… read more here.
Keywords: intrathecal onasemnogene; sitting nonambulatory; dose; onasemnogene abeparvovec ... See more keywords