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 and severe 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 th…
Matched conditions: CANAVAN DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Aspa Therapeutics • Aim: Disease control
Last updated May 20, 2026 11:55 UTC
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Massive leukodystrophy biobank aims to unlock secrets of rare brain diseases
Knowledge-focused Recruiting nowThis study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…
Matched conditions: CANAVAN DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 26, 2026 11:05 UTC