Wolfram syndrome

MONDO:0018105

Wolfram syndrome (WS) also known as DIDMOAD, is a neurodegenerative disorder characterized by type I diabetes mellitus (DM), diabetes insipidus (DI), sensorineural deafness (D), bilateral optical atrophy (OA) and neurological signs. Other related problems are urinary tract atony, ataxia, peripheral neuropathy, psychiatric disorders and/or seizures. 2 types of WS may be distinguished: type 1 and type 2 (WS1 and WS2).

Also known as: DIDMOAD, DIDMOAD syndrome, Wolfram syndrome, diabetes insipidus, diabetes mellitus, optic atrophy, and deafness syndrome, diabetes insipidus-diabetes mellitus-optic atrophy-deafness syndrome, diabetes mellitus and insipidus with optic atrophy and deafness

7 clinical trials for this condition and its sub-types, 7 tagged with Wolfram syndrome itself.

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Where it sits in the disease tree

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↑ Syndromic disease (7158)

Sub-types of Wolfram syndrome

Wolfram syndrome 1 1 trial
Wolfram syndrome 2 0 trials
Wolfram syndrome, mitochondrial form 0 trials

Including sub-types (7) Tagged with Wolfram syndrome (7)

Trials to join now! 2 Not yet recruiting 2 Not yet finished but already full! 2 Completed 1

Can a common drug slow vision loss in rare wolfram syndrome?

Disease control Completed

This study tested a drug called sodium valproate in 63 children and adults with Wolfram syndrome, a rare genetic disorder that causes vision loss and other health problems. Participants were randomly assigned to receive either the drug or a placebo to see if it could slow the wor…

Phase 2 • Sponsor: University of Birmingham • Aim: Disease control

Last updated Jun 27, 2026 12:29 UTC

Wolfram syndrome

Where it sits in the disease tree

Can a common drug slow vision loss in rare wolfram syndrome?