Wolfram Syndrome News and Research

RSS

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

A new Rare Diseases Action Plan for England will be published today (Tuesday 28 February) that will ensure those living with these conditions receive better care and treatment, fairer access to testing and have continued support.

28 Feb 2023

Why cheap, older drugs that might treat covid never get out of the lab

In March 2020, Dr. Joseph Vinetz left the contemplative world of his Yale University infectious-disease laboratory and plunged into the covid ward at Yale New Haven Hospital, joining an army of health care workers who struggled to treat the deadly viral disease.

19 Apr 2022

Yale researchers uncover potential treatment for lethal Wolfram Syndrome

Yale pharmacology professor Barbara Ehrlich and her team have uncovered a mechanism driving a rare, lethal disease called Wolfram Syndrome and also a potential treatment. Their findings appear in the July 6 edition of Proceedings of the National Academy of Sciences.

16 Jul 2020

Study: Patients’ genetically edited stem cells successfully reversed diabetes in mice

Using induced pluripotent stem cells produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes called Wolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used the gene-editing tool CRISPR-Cas9 to correct a genetic defect that had caused the syndrome.

22 Apr 2020

Doctoral thesis focuses on the role of oxidative stress in Wolfram syndrome and hypothermia

In the Faculty of Medicine at the University of Tartu, the first animal testings were conducted using antioxidant peptides designed and synthesized by scientists in Tartu, which may reduce oxidative stress.

29 May 2019

Newly designed molecule could benefit people with Friedrich's Ataxia

Skin cells taken from patients with a rare genetic disorder are up to ten times more sensitive to damage from ultraviolet A radiation in laboratory tests, than those from a healthy population, according to new research from the University of Bath.

21 Feb 2019

Researchers produce insulin-secreting cells from stem cells of diabetic patients

Signaling a potential new approach to treating diabetes, researchers at Washington University School of Medicine in St. Louis and Harvard University have produced insulin-secreting cells from stem cells derived from patients with type 1 diabetes.

11 May 2016

Dantrolene drug may be effective treatment for rare form of diabetes

A commonly prescribed muscle relaxant may be an effective treatment for a rare but devastating form of diabetes, researchers at Washington University School of Medicine in St. Louis report.

24 Nov 2014

Stem cells from Wolfram syndrome patients used to elucidate beta-cell failure pathway in diabetes

Scientists from the New York Stem Cell Foundation Research Institute and Columbia University Medical Center have used stem cells created from the skin of patients with a rare form of diabetes—Wolfram syndrome—to elucidate an important biochemical pathway for beta-cell failure in diabetes. The findings by Linshan Shang and colleagues were published today in Diabetes.

13 Nov 2013

Researchers identify gene mutations in Wolfram syndrome that affects insulin-secreting beta cells

The rare disorder Wolfram syndrome is caused by mutations in a single gene, but its effects on the body are far reaching. The disease leads to diabetes, hearing and vision loss, nerve cell damage that causes motor difficulties, and early death.

17 Sep 2012

TXNIP protein provides best available target for therapies against diabetes, Wolfram syndrome

Inflammation and cell stress play important roles in the death of insulin-secreting cells and are major factors in diabetes. Cell stress also plays a role in Wolfram syndrome, a rare, genetic disorder that afflicts children with many symptoms, including juvenile-onset diabetes.

8 Aug 2012