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Neurodevelopmental disorder with or without variable brain abnormalities; NEDBA

MONDO:0032755

Also known as: NEDBA, NEURODEVELOPMENTAL DISORDER WITH OR WITHOUT VARIABLE BRAIN ABNORMALITIES

1 clinical trial for this condition and its sub-types, 1 tagged with Neurodevelopmental disorder with or without variable brain abnormalities; NEDBA itself.

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↑ Mendelian neurodevelopmental disorder (207)

Including sub-types (1) Tagged with Neurodevelopmental disorder with or without variable brain abnormalities; NEDBA (1)

Not yet finished but already full! 1

Custom drug targets rare genetic brain condition in one patient

Disease control Ongoing

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed specifically for one person with a rare genetic brain disorder (NEDBA) caused by a MAPK8IP3 mutation. The goal is to see if the drug can improve motor skills and reduce seizures over 12 to 24 m…

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

Last updated Jun 27, 2026 07:52 UTC