Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.

doi:10.1056/NEJMoa2003715

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Article

Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.

The New England journal of medicine - 9 Jul 2020

Miller Timothy, Cudkowicz Merit, Shaw Pamela J, Andersen Peter M, Atassi Nazem, Bucelli Robert C, Genge Angela, Glass Jonathan, Ladha Shafeeq, Ludolph Albert L, Maragakis Nicholas J, McDermott Christopher J, Pestronk Alan, Ravits John, Salachas François, Trudell Randall, Van Damme Philip, Zinman Lorne, Bennett C Frank, Lane Roger, Sandrock Alfred, Runz Heiko, Graham Danielle, Houshyar Hani, McCampbell Alexander, Nestorov Ivan, Chang Ih, McNeill Manjit, Fanning Laura, Fradette Stephanie, Ferguson Toby A

Abstract excerpt

BACKGROUND: Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administration of tofersen is being studied for the treatment of amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. METHODS: We conducted a phase 1-2 ascending-dose trial evaluating tofersen in adults with ALS due to SOD1 mutations....

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PMID: 32640130
DOI: 10.1056/NEJMoa2003715

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