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Updated: Jul 12, 2026

Modification of the Treatment Methods for Wasting Marmoset Syndrome with Tranexamic Acid and Supportive Measures
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Published on: July 12, 2024

Consensus Recommendations for the Clinical Management of Wolfram syndrome Using a Delphi Method.

Josephine Elliott1, Saumel Ahmadi2, Patrick Yu-Wai-Man3,4,5,6

  • 1Department of Cancer and Genomic Sciences, University of Birmingham, United Kingdom.

Medrxiv : the Preprint Server for Health Sciences
|July 10, 2026
PubMed
Summary

These are the first international clinical guidelines for Wolfram syndrome, a rare neurodegenerative disorder. The consensus statements offer actionable recommendations for diagnosis, care, and treatment across multiple specialties.

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Last Updated: Jul 12, 2026

Modification of the Treatment Methods for Wasting Marmoset Syndrome with Tranexamic Acid and Supportive Measures
03:07

Modification of the Treatment Methods for Wasting Marmoset Syndrome with Tranexamic Acid and Supportive Measures

Published on: July 12, 2024

Area of Science:

  • Neuroscience
  • Genetics
  • Endocrinology

Background:

  • Wolfram syndrome is a rare, inherited neurodegenerative disorder, primarily caused by WFS1 gene mutations.
  • Characterized by diabetes mellitus, optic atrophy, hearing loss, and neurological deficits.
  • No approved disease-modifying treatments or established international clinical guidelines existed previously.

Purpose of the Study:

  • To establish the first international clinical consensus guidelines for Wolfram syndrome.
  • To provide actionable recommendations for the diagnosis, management, and multidisciplinary care of Wolfram syndrome patients.

Main Methods:

  • Systematic review of 273 publications by an international steering committee.
  • Modified three-round Delphi process involving international specialists across multiple disciplines.
  • Incorporated feedback from nurses, patient advocacy groups, and genetic diagnostic specialists.

Main Results:

  • 35 consensus statements achieved ≥80% agreement across key clinical domains.
  • Guidelines cover diagnosis, genetic testing, multidisciplinary care, and management of specific symptoms.
  • Recommendations span neuro-ophthalmology, neurology, endocrinology, urology, gastroenterology, and psychiatry.

Conclusions:

  • These guidelines represent the first international clinical consensus for Wolfram syndrome.
  • Provide actionable recommendations for clinicians globally.
  • Implementation should include prospective audits to refine future guidelines.