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Congenital disorder of deglycosylation

MONDO:0031376

2 clinical trials for this condition and its sub-types.

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Sub-types

Congenital disorder of deglycosylation 1 (2) Congenital disorder of deglycosylation 2 (0)

Broader categories

Disease (680) Metabolic disease (233) Hereditary disease (176) Inborn errors of metabolism (45) Human disease (14) Disease of genetic or genomic mechanism (2) Disease by developmental or physiological process (0) Disease by etiologic mechanism (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: PHASE3 • Sponsor: Grace Science, LLC • Aim: Disease control

Last updated Jun 27, 2026 12:29 UTC