Records containing: NGLY1 Deficiency
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CLINICAL_TRIALS study record update · 2025-07-08A Phase II Randomized, Multicenter, Double-Blind, Placebo-Controlled Study Evaluating Effect Of GlcNAc On Tear Production In Individuals With NGLY1-CDDG
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CLINICAL_TRIALS study record update · 2025-04-24A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency
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CLINICAL_TRIALS study record update · 2025-04-11Investigation of NGLY1 Deficiency Movement Disorder & Clinical Features