WS Research 2021 - 2022

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• Two Cases of Wolfram Syndrome Who Were Initially Diagnosed With Type 1 Diabetes. May, 2022

 

• ,Loss of Function of WFS1 Causes ER Stress-Mediated Inflammation in Pancreatic Beta-Cells.  Morikawa et al. Frontiers in Endocrinology.  March, 2022.

 

• Activation of the sigma-1 receptor chaperone alleviates symptoms of Wolfram syndrome in preclinical models. Feb 2022

 

• Wolfram Syndrome 1: From Genetics to Therapy.   2022

 

• Case Report: Off-Label Liraglutide Use in Children With Wolfram Syndrome Type 1: Extensive Characterization of Four Patients. December, 2021.

 

• Early Intervention and Lifelong Treatment with GLP1 Receptor Agonist Liraglutide in a Wolfram Syndrome Rat Model with an Emphasis on Visual Neurodegeneration, Sensorineural Hearing Loss and Diabetic Phenotype.  November, 2021.

 

• Multiomic analysis on human cell model of wolfram syndrome reveals changes in mitochondrial morphology and function. November, 2021.

 

• ​Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience. November, 2021.

 

• Unique three-site compound heterozygous mutation in the WFS1 gene in Wolfram syndrome.  August, 2021.


• Wolfram syndrome: new pathophysiological insights and therapeutic strategies. August, 2021.


• A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome June, 2021.

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• Clinical Spectrum Associated with Wolfram Syndrome Type 1 and Type 2: A Review on Genotype–Phenotype Correlations.    April, 2021.

 

• Liraglutide, 7,8-DHF and their co-treatment prevents loss of vision and cognitive decline in a Wolfram syndrome rat model Jan, 2021.​