Current Landscape of Treatments for Wolfram Syndrome

Trends Pharmacol Sci. 2019 Oct;40(10):711-714. doi: 10.1016/j.tips.2019.07.011. Epub 2019 Aug 13.

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.

Keywords: WFS1; Wolfram syndrome; endoplasmic reticulum; gene therapy; regenerative therapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Genetic Therapy / methods*
  • Humans
  • Molecular Targeted Therapy
  • Regenerative Medicine / methods*
  • Wolfram Syndrome / drug therapy
  • Wolfram Syndrome / genetics
  • Wolfram Syndrome / metabolism
  • Wolfram Syndrome / therapy*