Glutaric aciduria
MONDO:0000129Also known as: glutaric acidemia, glutaric aciduria, glutaric aciduria (disease)
5 clinical trials for this condition and its sub-types, 0 tagged with Glutaric aciduria itself.
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Sub-types of Glutaric aciduria
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5 sub-types
- Glutaric acidemia IIa 0 trials
- Glutaric acidemia IIb 0 trials
- Glutaric acidemia IIc 0 trials
- Multiple acyl-CoA dehydrogenase deficiency, mild type 0 trials
- Multiple acyl-CoA dehydrogenase deficiency, severe neonatal type 0 trials
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Glutaric acidemia type 3 0 trials
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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
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Can tracking MADD's natural course unlock better care?
Knowledge-focused Recruiting nowThis study follows people with Multiple Acyl-CoA Dehydrogenase Deficiency (MADD) over time to learn how the condition progresses and affects daily life. Researchers will collect health data and patient feedback to identify patterns and potential markers of the disease. The goal i…
Sponsor: Icahn School of Medicine at Mount Sinai • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC