Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study.

doi:10.1007/s13311-022-01237-4

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Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics - 1 Jul 2022

Benatar Michael, Wuu Joanne, Andersen Peter M, Bucelli Robert C, Andrews Jinsy A, Otto Markus, Farahany Nita A, Harrington Elizabeth A, Chen Weiping, Mitchell Adele A, Ferguson Toby, Chew Sheena, Gedney Liz, Oakley Sue, Heo Jeong, Chary Sowmya, Fanning Laura, Graham Danielle, Sun Peng, Liu Yingying, Wong Janice, Fradette Stephanie

Abstract excerpt

Despite extensive research, amyotrophic lateral sclerosis (ALS) remains a progressive and invariably fatal neurodegenerative disease. Limited knowledge of the underlying causes of ALS has made it difficult to target upstream biological mechanisms of disease, and therapeutic interventions are usually administered relatively late in the course of disease. Genetic forms of ALS offer a unique opportunity for...

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PMID: 35585374
DOI: 10.1007/s13311-022-01237-4
PMCID: PMC9587202

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