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Gene Therapy in Amyotrophic Lateral Sclerosis.

Cells · 2022 · PMC9265980 · PMID 35805149

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However, since participant numbers were low in the initial trial, BIIB067 was extended to a phase 3 clinical trial; however, the primary outcomes of measuring disease progression in ALS fast progressors at 28 weeks of treatment did not reach statistical significance [ 36 ].

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