Records containing: Mutation in the Superoxide Dismutase-1 (SOD1) Gene

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CLINICAL_TRIALS study record update · 2026-02-27
First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations