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GS-100 NGLY1 gene therapy

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Phase 1/2/3 program of AAV9-mediated NGLY1 gene transfer in children and adolescents with NGLY1 deficiency.

NCT ID
NCT06199531
Sponsor
Grace Science, LLC
Phase
Phase 3
Indication
NGLY1 deficiency; impaired removal of N-glycans from misfolded proteins
Started
2024-02-13
Status
Active, not recruiting