Current Landscape of Treatments for Wolfram Syndrome

Damien Abreu, Fumihiko Urano

Research output: Contribution to journalShort surveypeer-review

9 Scopus citations

Abstract

Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.

Original languageEnglish
Pages (from-to)711-714
Number of pages4
JournalTrends in Pharmacological Sciences
Volume40
Issue number10
DOIs
StatePublished - Oct 2019

Keywords

  • WFS1
  • Wolfram syndrome
  • endoplasmic reticulum
  • gene therapy
  • regenerative therapy

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