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Inborn aminoacylase deficiency
MONDO:0017686An inherited metabolic disease that is has its basis in the disruption of aminoacylase activity.
Also known as: inborn aminoacylase activity disorder, inborn error of aminoacylase activity, rare inborn error of aminoacylase activity, aminoacylase deficiency
6 clinical trials for this condition and its sub-types, 0 tagged with Inborn aminoacylase deficiency itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Inborn aminoacylase deficiency
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Canavan disease 6 trials
2 sub-types
- Mild Canavan disease 0 trials
- Severe Canavan disease 0 trials
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Aminoacylase 1 deficiency 0 trials
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Experimental gene therapy hopes to slow rare brain disease in toddlers
Disease control Recruiting nowThis study tests a gene therapy called BBP-812 in children up to 30 months old with Canavan disease, a rare genetic brain disorder. The treatment uses a harmless virus to deliver a working copy of the ASPA gene, aiming to reduce harmful brain chemicals and improve motor and think…
Phase 1/2 • Sponsor: Aspa Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:09 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC