Aspartylglucosaminuria

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CLINICAL_TRIALS trial_result · 2026-04-15
Clinical Trial Update: Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU) [Phase 1, Phase 2]
Phase 1/2 clinical trial evaluating scAAV9/AGA gene therapy for Aspartylglucosaminuria, with recruitment not yet started.