Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Timothy MillerMerit CudkowiczPamela J ShawPeter Munch AndersenNazem AtassiRobert C BucelliAngela GengeJonathan GlassShafeeq LadhaAlbert L LudolphNicholas J MaragakisChristopher J McDermottAlan PestronkJohn RavitsFrançois SalachasRandall TrudellPhilip Van DammeLorne ZinmanC Frank BennettRoger LaneAlfred SandrockHeiko RunzDanielle GrahamHani HoushyarAlexander McCampbellIvan NestorovIh ChangManjit McNeillLaura FanningStephanie FradetteToby A FergusonPublished in: The New England journal of medicine (2020)
In adults with ALS due to SOD1 mutations, CSF SOD1 concentrations decreased at the highest concentration of tofersen administered intrathecally over a period of 12 weeks. CSF pleocytosis occurred in some participants receiving tofersen. Lumbar puncture-related adverse events were observed in most participants. (Funded by Biogen; ClinicalTrials.gov number, NCT02623699; EudraCT number, 2015-004098-33.).