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Disease

Wolfram Syndrome

Active therapeutic pipelineEmerging research
1
Publications
4
Clinical trials
2023
Latest publication
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

Genomics of Wolfram Syndrome 1 (WFS1).

Research2023-09-04Biomolecules

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

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Clinical trials

4 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
4
All trials
1
Active
0
Late-stage
2
Completed

Research activity

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Most influential

Genomics of Wolfram Syndrome 1 (WFS1).

Biomolecules · 2023 · 28 cites
Recent publications

Genomics of Wolfram Syndrome 1 (WFS1).

Biomolecules · 2023 · 28 cites
Major themes2
  • Optic Atrophy1
  • Wolfram Syndrome1
Leading journals1
  • Biomolecules1
Leading researchers1
  • Kõks S1
Affiliations (unnormalised)2
  • Centre for Molecular Medicine and Innovative Therapeutics1
  • Perron Institute for Neurological and Translational Science1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A hereditary condition characterized by multiple symptoms including those of DIABETES INSIPIDUS; DIABETES MELLITUS; OPTIC ATROPHY; and DEAFNESS. This syndrome is also known as DIDMOAD (first letter of each word) and is usually associated with VASOPRESSIN deficiency. It is caused by mutations in gene WFS1 encoding wolframin, a 100-kDa transmembrane protein.

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.