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A Phase 1/2 Open-Label Intrathecal Administration of TSHA-105 to Determine the Safety and Efficacy in 2 Subjects With SLC13A5 Citrate Transporter Disorder Caused by a Mutation in the SLC13A5 Gene

Phase 1/2, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of TSHA-105 in individuals with SLC13A5 Citrate Tra…