TUBB4A-related neurologic disorder
MONDO:0800470Any neurologic condition in which the cause of the disease is a mutation in the TUBB4A gene.
4 clinical trials for this condition and its sub-types, 4 tagged with TUBB4A-related neurologic disorder itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of TUBB4A-related neurologic disorder
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Hypomyelinating leukodystrophy 6 2 trials
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Custom-Made drug targets rare genetic brain disorder in Single-Child trial
Disease control By invitation onlyThis 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…
Phase 1/2 • 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…
Phase 1/2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC