Wolfram syndrome
MONDO:0018105Wolfram 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.
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Broader categories
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Hope for rare wolfram syndrome: drug aims to slow diabetes progression
Disease control OngoingThis study tests an experimental drug called AMX0035 in 12 adults with Wolfram syndrome, a rare genetic disorder that causes diabetes and vision loss. The goal is to see if the drug can help preserve the pancreas's ability to make insulin and to check its safety over up to 4 year…
Phase: PHASE2 • Sponsor: Amylyx Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Gene study aims to solve wolfram syndrome mystery
Knowledge-focused OngoingThis study looks at 45 people with certain changes in the WFS1 gene to figure out if they have a milder, dominant form of wolfram-like syndrome instead of the more severe recessive form. Researchers will use eye scans (OCT) to see if the gene changes are linked to specific eye pr…
Sponsor: Hôpital Necker-Enfants Malades • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:03 UTC