TUBB4A-related neurologic disorder
MONDO:0800470Any neurologic condition in which the cause of the disease is a mutation in the TUBB4A gene.
17 clinical trials for this condition and its sub-types.
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Broader categories
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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…
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…
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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AI boosts brain scan accuracy in massive new trial
Diagnosis ENROLLING_BY_INVITATIONThis study tests whether an AI tool can help radiologists read brain CT and MRI scans more accurately and quickly. Researchers will compare how well doctors, AI alone, and doctors using AI together can spot abnormalities, urgent findings, and classify diseases. The goal is to red…
Sponsor: Yaou Liu • Aim: Diagnosis
Last updated Jun 27, 2026 11:00 UTC
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Could a headset at home boost brain recovery? new study tests tDCS for stroke, tumors, and neurodegeneration
Symptom relief ENROLLING_BY_INVITATIONThis study tests whether a home-based brain stimulation device (tDCS) combined with activity therapy can help improve cognitive and language problems in people with stroke, brain tumors, or neurodegenerative conditions like Parkinson's or Alzheimer's. Fifty-five participants will…
Phase: NA • Sponsor: Mayo Clinic • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC