Abstract
The Food and Drug Administration (FDA) have approved disease modifying therapies that are changing the management of Spinal Muscular Atrophy (SMA). Nusinersen is an antisense oligonucleotide that increases production of SMN protein by splicing SMN2 pre-mRNA. It was approved by the FDA for use in all types of SMA in December of 2016. AVXS-101 is a gene replacement strategy with AAV9 and was approved in May of 2019 for use in children younger than two years of age. We describe four patients with SMA type 1 who have been treated in our facility with Nusinersen followed by AVXS-101.
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