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A cure for Wolfram syndrome could lead to a cure for diabetes and blindness.

Tuesday, January 7, 2014

My goal in 2014, a small scale interventional study for Wolfram syndrome

My goal this year is to start a small scale interventional study for Wolfram syndrome. I believe that conducting a clinical trial in a small group of patients with Wolfram syndrome of homogeneous etiology could potentially lead to a breakthrough in treatments for other conditions associated with endoplasmic reticulum dysfunction, including type 1 diabetes and neurodegeneration. We all should be aware and appreciate the power of rare diseases, such as Wolfram syndrome. I was recently asked to write my view on type 1 diabetes and Wolfram syndrome by Nature, and this was just published. I hope... sincerely hope that this article will raise awareness of Wolfram syndrome.