TUBB4A-RELATED LEUKODYSTROPHY
Clinical trials for TUBB4A-RELATED LEUKODYSTROPHY explained in plain language.
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Custom-Made drug targets rare genetic brain disorder in Single-Child trial
Disease control ENROLLING_BY_INVITATIONThis study tests a personalized drug called nL-TUBB4-001, designed specifically for one child with TUBB4A-related leukodystrophy, a rare genetic disease that damages the brain. The drug is an antisense oligonucleotide, which aims to correct the genetic error. The trial will monit…
Matched conditions: TUBB4A-RELATED LEUKODYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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One-Person trial aims to tackle rare genetic brain disease
Disease control OngoingThis study tests a custom-made drug called an antisense oligonucleotide in a single person with TUBB4A-related leukodystrophy, a rare genetic disorder that affects the brain's white matter. The drug is designed to target the specific genetic mutation causing the disease. Research…
Matched conditions: TUBB4A-RELATED LEUKODYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC