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Leukodystrophy, demyelinating, adult-onset

MONDO:0976138

3 clinical trials for this condition and its sub-types.

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Sub-types

Adult-onset autosomal dominant demyelinating leukodystrophy (3) Leukodystrophy, demyelinating, adult-onset, autosomal dominant, atypical (0) Leukodystrophy, demyelinating, adult-onset, autosomal dominant, typical (0)

Broader categories

Disease (717) Nervous system disorder (243) Hereditary disease (188) Neurodegenerative disease (182) Central nervous system disorder (115) Human disease (15) Inherited neurodegenerative disorder (10) Hereditary neurological disease (6) Leukodystrophy (5) Disease of genetic or genomic mechanism (2)
Trials to join now! 1 Not yet finished but already full! 1 Completed 1
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  • Custom-Made genetic drug targets rare brain disorder in one patient

    Disease control Ongoing

    This study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with autosomal dominant leukodystrophy (ADLD), a rare genetic brain disease. The treatment aims to slow or stop the disease by targeting the underlying genetic mutat…

    Phase 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control

    Last updated Jul 15, 2026 00:00 UTC

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