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NGLY1 Deficiency
Clinical trial pipeline · Data from ClinicalTrials.gov
See which NGLY1 Deficiency trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which NGLY1 Deficiency trials you may qualify forA non-randomized, open-label, Phase 1/2/3 study of a single intracerebroventricular (ICV) administration of a gene replacement therapy (GS-100) in participants…
In patients with NGLY1-CDDG, the disorder can lead to eye damage due to not being able to produce enough tears. This study is being done to see if the dietary s…
N-glycanase 1 (NGLY1) Deficiency (OMIM #615273) is an ultra-rare, autosomal recessive disorder caused by loss of function variants in NGLY1 gene. The multisyste…
NGLY1 deficiency is a rare genetic disorder that is characterized by: global developmental delay and/or intellectual disability, hypo- or alacrima, transient el…