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Congenital disorder of deglycosylation

MONDO:0031376

2 clinical trials for this condition and its sub-types, 0 tagged with Congenital disorder of deglycosylation itself.

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Where it sits in the disease tree

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Part of

↑ Inborn errors of metabolism (2228)

Sub-types of Congenital disorder of deglycosylation

Congenital disorder of deglycosylation 1 2 trials
Congenital disorder of deglycosylation 2 0 trials

Including sub-types (2) Tagged with Congenital disorder of deglycosylation (0)

Trials to join now! 1 Not yet finished but already full! 1

Experimental gene therapy aims to halt rare childhood disease

Disease control Ongoing

This study tests a one-time gene therapy called GS-100 in 10 children aged 2 to 18 with NGLY1 deficiency, a rare genetic disorder. The therapy is given directly into the brain fluid to deliver a working copy of the missing gene. The goal is to improve motor skills and development…

Phase 3 • Sponsor: Grace Science, LLC • Aim: Disease control

Last updated Jun 27, 2026 12:29 UTC