Wilson's Disease News and Research

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Penicillamine challenge ineffective for detecting Wilson's disease in asymptomatic children

An Italian research team confirmed that the scoring system for Wilson's disease (WD) provides good diagnostic accuracy with 93% positive and 92% negative predictive values, respectively in children with mild liver disease. In asymptomatic children, a urinary copper excretion above 40 μg/24 hours was suggestive of WD, however the penicillamine challenge test (PCT) did not provide an accurate diagnosis in this patient subset.

1 Dec 2010

FDA extends review on Acetadote for non-acetaminophen acute liver failure

Cumberland Pharmaceuticals Inc. today announced the U.S. Food and Drug Administration (FDA) has extended its review of the supplemental new drug application (sNDA) for the use of Acetadote® (acetylcysteine) Injection in patients with non-acetaminophen acute liver failure.

21 Aug 2010

Intellect Neurosciences locks database of OXIGON Phase 1b clinical trial for Alzheimer's disease

Intellect Neurosciences, Inc. a biopharmaceutical company with an internal preclinical and clinical-stage pipeline and licenses with major pharmaceutical companies covering products in late-stage clinical trials, announced that it has locked the database of the Company's Phase 1b clinical trial for its lead Alzheimer's candidate, OXIGON™ (OX1).

18 Aug 2010

St. Luke's joins Dr. John Goss in celebration of his 1,000th liver transplantation procedure

St. Luke's Episcopal Hospital (St. Luke's) joins John Goss, M.D., in celebrating his milestone as he recently performed his 1,000th liver transplantation procedure. The largest liver transplant program in Houston's world-renowned Texas Medical Center, St. Luke's performs approximately 75 liver transplants annually. Additionally, St. Luke's liver transplant survival rate is the highest in the nation, with a one-year adult patient survival rate of 96.5 percent compared to the national expected survival rate of 89.38 percent.

24 Jun 2010

Liver biopsy with hepatic copper quantification every 3 years needs to be considered for WD patients

As a result of the rarity of Wilson disease (WD) and the fact that liver biopsy is not performed routinely during follow-up, unless clinically indicated, the progression and timing of the liver pathology and its correlation with different anti-copper treatments or aminotransferase levels are poorly characterized. Previous studies have demonstrated the possibility of improvement of steatosis and inflammation grade, and of fibrosis stage during long-term follow-up.

12 Apr 2010

Copper sequestering drug tetrathiomolybdate shown to be effective in treating Wilson disease

The copper sequestering drug tetrathiomolybdate (TM) has been shown in studies to be effective in the treatment of Wilson disease, a disease caused by an overload of copper, and certain metastatic cancers.

27 Nov 2009

Comparison study finds no significant differences in post-transplant survival rates of NASH and ETOH groups

Researchers at the University of Miami School of Medicine compared the outcomes of cirrhotic patients who underwent liver transplants for nonalcoholic steatohepatitis (NASH) versus alcoholic liver disease (ETOH) and found no statistically significant differences in post-transplant survival rates between the NASH and ETOH groups

25 Nov 2009

Wilson disease and excess copper are focus of new NIDDK publication

Wilson disease, a rare but potentially serious condition that causes the buildup of too much copper in the body, is explained in a new fact sheet from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at the National Institutes of Health (NIH).

1 Jun 2009

Scientists discover how mutation in the copper-regulating protein ATP7B causes Wilson disease

Using a combination of computer simulations and cutting-edge lab experiments, physical biochemists at Rice University have discovered how a small genetic mutation -- which is known to cause Wilson disease -- subtly changes the structure of a large, complex protein that the body uses to keep copper from building up to toxic levels.

19 Aug 2008

Wilson's Disease News and Research

Penicillamine challenge ineffective for detecting Wilson's disease in asymptomatic children

FDA extends review on Acetadote for non-acetaminophen acute liver failure

Intellect Neurosciences locks database of OXIGON Phase 1b clinical trial for Alzheimer's disease

St. Luke's joins Dr. John Goss in celebration of his 1,000th liver transplantation procedure

Liver biopsy with hepatic copper quantification every 3 years needs to be considered for WD patients

Copper sequestering drug tetrathiomolybdate shown to be effective in treating Wilson disease

Comparison study finds no significant differences in post-transplant survival rates of NASH and ETOH groups

Wilson disease and excess copper are focus of new NIDDK publication

Scientists discover how mutation in the copper-regulating protein ATP7B causes Wilson disease

Australian researcher unveils pregnancy mystery

Drug shortfall as more than 5,000 conditions need medication

5,000 rare diseases need drugs, but Europe only approves a handful each year

New study to determine the effectiveness of a new pediatric screening method for Wilson's disease

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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