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BioWorld - Sunday, February 22, 2026
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Home » More stem cell, gene therapies progressing in clinic for ALS
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Rare Genetic Disease Symposium

More stem cell, gene therapies progressing in clinic for ALS

Sep. 23, 2025
By Marian (YoonJee) Chu
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Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disorder with no known cure. While three therapies have gained U.S. FDA approvals to date, including Rilutek (riluzole), Radicava/Radicava ORS (edaravone) and tofersen (BIIB-067, the lack of a disease-modifying drug has spurred the continual search for novel therapies.
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