Publication: biomedcentral.com | Publication Date: 06 March 2025
Authors: Jagomäe, T., Velling, S., Tikva, T.B. et al.
Wolfram syndrome (WS) is a rare autosomal disorder caused by WFS1 gene mutations, currently lacking approved treatments. Preclinical and clinical reports suggest that diabetes medications, such as glucagon-like peptide-1 receptor agonist (GLP1-RA), slow WS-related diabetes and neurodegeneration, improving patient outcomes.
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Publication: science.org | Publication Date: 2025
Authors: Kikuko Amo-Shiinoki et al.
Insulin-dependent diabetes in patients with Wolfram syndrome (WS; OMIM 222300) has been linked to endoplasmic reticulum (ER) stress caused by WFS1 gene mutations. However, the pathological process of ER stress–associated β cell failure remains to be fully elucidated. Our results indicate loss of β cell lineage and subsequent dedifferentiation as the mechanisms underlying functional and mass deficits in WS.
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Join us for a great event on September 8, 2025 at Bogey Hills Country Club to support The Snow Foundation. The day will feature, 10 Par 3 holes with unique hole in on contests, 4 Par 4’s and 4 Par 5 Long Drive holes, a $100,000 Shootout for 10 Golfers.
Download the flyer for registration and sponsorship opportunities.
June 13 & 14th 2025, Paris, France
The 10th International Congress of Clinicians and Researchers aims to evaluate current research efforts, present the results of the TreatWolfram clinical trial, and assess medical care.
Diabetes is an endocrine disorder that affects the way your pancreas produces insulin and later glucose distribution. Diabetes is a chronic condition involving issues regulating blood sugar (glucose). When glucose levels are too high, you may be at risk of developing problems with your kidneys, heart, and other parts of your body.
https://www.healthline.com/health/is-diabetes-an-endocrine-disorder
Cherney, Kristeen. (2024, October 3). Is Diabetes Considered an Endocrine Disorder? Healthline.
A groundbreaking gene therapy treatment has shown remarkable success in improving vision for patients with Leber congenital amaurosis (LCA1), a rare inherited condition that causes severe vision loss. The therapy, which targets a specific genetic mutation, has resulted in significant improvements in vision for some patients.
https://www.vanguardngr.com/2024/09/gene-therapy-offers-hope-for-patients-with-rare-vision-loss/
Ogundipe, Sola. (2024, September 29). Gene therapy offers hope for patients with rare vision loss. Vanguard.
Quality measures are a tool to drive healthcare toward value-based care, and in diabetes care these measures can improve diabetes management and prevent downstream conditions, such as cardiovascular disease. However, there are multiple hurdles that healthcare organizations face when implementing quality measures for diabetes care, explained Sean M. Oser, M.D., M.P.H., associate professor in the Department of Family Medicine at the University of Colorado School of Medicine, in a Managed Healthcare Executive K-Cast video series.
MHE Staff (2024, October 3). Diabetes Quality Measures Have Challenges But Also Successes. Managed Healthcare Executive.
Diseases that affect the retina, the light-sensitive layer at the back of the eye, are a significant cause of visual impairment and blindness. Gene therapy holds promise for treating some of these conditions, and current research advances may soon shift the therapeutic landscape for eye health. However, many obstacles remain in place, as this Special Feature discusses.
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
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