AI Article Synopsis
- Wolfram syndrome is a rare genetic disorder that includes symptoms like insulin-dependent diabetes, optic nerve damage, and neurological decline.
- Current treatments do not exist to slow down or reverse the disease's progression, highlighting the need for new therapies.
- The text discusses plans for developing innovative treatment strategies aimed at finding a cure for Wolfram syndrome.
Article 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.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7547529 | PMC |
| http://dx.doi.org/10.1016/j.tips.2019.07.011 | DOI Listing |
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