CANAVAN DISEASE
Clinical trials for CANAVAN DISEASE explained in plain language.
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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…
Matched conditions: CANAVAN DISEASE
Phase: PHASE1, PHASE2 • 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…
Matched conditions: CANAVAN DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC