DNL343 and Disease Progression in Amyotrophic Lateral Sclerosis

In this randomized clinical trial, despite the relevance of the eIF2b activation pathway in ALS disease biology and supporting evidence of proof of mechanism and optimal dose selection in a prior phase 1B ALS clinical trial, 200-mg/d DNL343 did not show evidence of slowing disease progression in ALS, highlighting the need for alternative therapeutic approaches.

Corresponding Author: Sabrina Paganoni, MD, PhD, Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, 165 Cambridge St, Ste 600, Boston, MA 02114 (spaganoni@mgh.harvard.edu).

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