Glutaryl-CoA dehydrogenase deficiency
MONDO:0009281Glutaryl-CoA dehydrogenase (GCDH) deficiency (GDD) is an autosomal recessive neurometabolic disorder clinically characterized by encephalopathic crises resulting in striatal injury and a severe dystonic dyskinetic movement disorder.
Also known as: GA1, GCDHD, glutaric acidemia type 1, glutaric aciduria type 1, glutaric aciduria, type 1, glutaricaciduria, type I, glutaryl-CoA dehydrogenase deficiency, glutaryl-coenzyme A dehydrogenase deficiency
4 clinical trials for this condition and its sub-types, 4 tagged with Glutaryl-CoA dehydrogenase deficiency itself.
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Experimental gene therapy offers hope for fatal nerve disease
Disease control Recruiting nowThis study tests a gene therapy for GM1 gangliosidosis, a fatal disorder that destroys nerve cells. The therapy uses a harmless virus to deliver a working gene, helping the body produce a missing enzyme. Up to 54 children with Type I (ages 6-12 months) or Type II (ages 1-12 years…
Phase 1/2 • Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Disease control
Last updated Sep 13, 2026 00:00 UTC
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New gene therapy trial hopes to tame rare metabolic disease in kids
Disease control Recruiting nowThis early-stage trial tests a one-time gene therapy called VGM-R02b in up to 12 children aged 6 years or younger with Glutaric Acidemia Type I, a rare genetic disorder that can cause brain damage. The study aims to see if the treatment is safe and can improve symptoms like movem…
Phase 1 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC