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Article

Onasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy

1 Jan 2021

Abstract excerpt

Spinal muscular atrophy (SMA) is a debilitating disorder characterized by degeneration of large motor neurons. It is a heterogeneous group of disorders caused by a homozygous deletion in the survival motor neuron (SMN) gene on chromosome 5, resulting in a SMN protein deficiency. Small amounts of SMN protein are also produced by the SMN2 gene, which that differs from SMN1 by a single nucleotide. Spinal muscular...

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PMID: 34239394
DOI: 10.5863/1551-6776-26.5.437

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