X-LINKED ADRENOLEUKODYSTROPHY
Clinical trials for X-LINKED ADRENOLEUKODYSTROPHY explained in plain language.
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Experimental gene therapy aims to halt fatal brain disease in children
Disease control Recruiting nowThis trial tests a gene therapy for X-linked adrenoleukodystrophy (X-ALD), a rare genetic disease that damages the brain. The therapy uses a modified virus to deliver a working copy of the faulty gene directly into the spinal fluid and bloodstream. Up to 30 patients aged 1 year a…
Matched conditions: X-LINKED ADRENOLEUKODYSTROPHY
Phase: NA • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Jun 26, 2026 14:43 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: X-LINKED ADRENOLEUKODYSTROPHY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC