Ngly1 Deficiency
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CLINICAL_TRIALS research · 2025-07-08Clinical Trial Update: A Study of GlcNAc on Tear Production in NGLY1-CDDG [Phase 2]
A Phase 2 clinical trial evaluating N-acetylglucosamine (GlcNAc) for tear production in individuals with NGLY1 deficiency. The study is curr…
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CLINICAL_TRIALS research · 2025-04-24Clinical Trial Update: Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency [Phase 1, Phase 2]
This clinical trial update concerns the safety and potential efficacy of GS-100 Gene Therapy for NGLY1 Deficiency in Phase 1/2 and indicates…
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CLINICAL_TRIALS research · 2025-04-11Clinical Trial Update: NGLY1 Natural History
The Baylor College of Medicine–led NGLY1 Deficiency natural history clinical trial (NCT06122766) has completed.