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A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated
Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase
(OTC) in Patients with Late-Onset OTC Deficiency

This study has been transitioned to CTIS with ID 2024-514337-38-00 check the CTIS register for the current data. PrimaryTo evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecondaryTo…

Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study

This study has been transitioned to CTIS with ID 2024-516124-34-00 check the CTIS register for the current data. Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3)…