Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type I

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Funded by AHRQ K08 HS026510-01A1 (to A.F.). A.V. received personal compensation for serving on advisory boards for AveXis, Biogen, and PTC therapeutics and as speaker for AveXis; served as investigator for AVXS-101 Managed access program; and serves as an Associate Editor for Medlink Neurology. J.P. serves as an investigator/advisor for AveXis, Biogen, Scholar Rock, Sarepta, PTC Therapeutics, and Genentech. The other authors declare no conflicts of interest.

Portions of this study were presented at the American Academy of Neurology annual meeting, << >>, 2020, << >>; and at the Muscular Dystrophy Association clinical and scientific conference, << >>, 2020, << >>.

© 2020 Elsevier Inc. All rights reserved.

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