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
Issue number10
StatePublished - Oct 2019


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


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