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This is an observational study following the natural course of NGLY1 deficiency over time. Individuals aged 2-25 with a molecularly confirmed diagnosis of NGLY1 deficiency took part. It has no assigned phase and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: \- Individuals aged 2 to 25 years with a confirmed molecular diagnosis of NGLY1 Deficiency. Exclusion Criteria: \- Prior or current participation in a therapeutic trial for NGLY1 Deficiency (note that neither the plan for future participation in an interventional trial nor participation in previous pilot study preclude entering this trial).