CHMP Backs Orphan Hybrid Drug for Wilson's Disease CHMP Backs Orphan Hybrid Drug for Wilson's Disease

Trientine tetrahydrochloride (Cuprior) is indicated for treatment of Wilson's disease in adults, adolescents, and children aged 5 years and older who are intolerant to D-penicillamine.International Approvals
Source: Medscape Pharmacist Headlines - Category: Drugs & Pharmacology Tags: Gastroenterology News Alert Source Type: news

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We report a pediatric case of D-penicillamine induced ANCA-associated vasculitis that manifests as a pulmonary-renal syndrome with a mild renal manifestation. A 13-year-old girl who has been taking D-penicillamine for five years under the diagnosis of Wilson disease visited the emergency room because of hemoptysis and dyspnea. She had diffuse pulmonary hemorrhage, microscopic hematuria, and proteinuria. Myeloperoxidase ANCA was positive, and a renal biopsy revealed pauci-immune crescentic glomerulonephritis. Under the diagnosis of D-penicillamine-induced ANCA-associated vasculitis, D-penicillamine was switched to trientine...
Source: Journal of Korean Medical Science - Category: Biomedical Science Tags: J Korean Med Sci Source Type: research
Authors: Mavilia MG, Sharma A, Forouhar F Abstract Cholangiocarcinoma (CC) is the second most common primary hepatic malignancy. Although the frequency of malignancy is generally increased in chronic liver disease, CC rarely presents in Wilson disease (WD). The incidence of hepatic malignancy in WD is only 1.2%, with CC accounting for 0.5%. A 66 year old male with history of hypertension, diabetes, and compensated cryptogenic cirrhosis presented with acute onset dyspnea and pleuritic chest pain. He was incidentally found to have a sizeable mass in the right hepatic lobe. Ultimately, a liver biopsy revealed dense de...
Source: Annals of Hepatology - Category: Gastroenterology Tags: Ann Hepatol Source Type: research
AbstractIdiopathic copper toxicosis (ICT) is characterized by marked copper deposition, Mallory-Denk body (MDB) formation and severe hepatic injury. Although the characteristics are apparently different from Wilson disease, large amounts of copper accumulate in the liver of the patients. We extensively treated a patient with ICT to reduce the body copper, however, the patient needed liver transplantation. Previous liver biopsy revealed high copper content. But extirpated liver contained an extremely small amount of copper, although MDBs and severe inflammation remained. These phenomena suggest abnormal copper metabolism is...
Source: Medical Molecular Morphology - Category: Molecular Biology Source Type: research
Authors: Saba L, Tiwari A, Biswas M, Gupta SK, Godia-Cuadrado E, Chaturvedi A, Turk M, Suri HS, Orru S, Sanches JM, Carcassi C, Marinho RT, Asare CK, Khanna NN, B K M, Suri JS Abstract Wilson's disease (WD) is an autosomal recessive disorder which is caused by poor excretion of copper in mammalian cells. In this review, various issues such as effective characterization of ATP7B genes, scope of gene network topology in genetic analysis, pattern recognition using different computing approaches and fusion possibilities in imaging and genetic dataset are discussed vividly. We categorized this study into three major sec...
Source: Frontiers in Bioscience - Elite - Category: Biomedical Science Tags: Front Biosci (Elite Ed) Source Type: research
Publication date: Available online 21 May 2019Source: Brain StimulationAuthor(s): Hu Liang Low, Sian K. Alexander, Anjum Misbahuddin, Godfrey T. Gillett
Source: Brain Stimulation - Category: Neurology Source Type: research
Publication date: Available online 24 May 2019Source: Journal of Trace Elements in Medicine and BiologyAuthor(s): Şükrü Güngör, Mukadder Ayşe Selimoğlu, Fatma İlknur Varol, Serdal Güngör, Muhammed Mehdi ÜremişAbstractObjectivesWilson’s disease (WD) is a metabolic disorder leading to hepatic and extrahepatic copper deposition. Several studies have reported that besides copper (Cu), iron (Fe) and zinc (Zn) are also accumulated at varying levels in various tissues in WD. However, there is not an adequate number of studies investigating the effects of Fe and Zn status on WD present...
Source: Journal of Trace Elements in Medicine and Biology - Category: Biochemistry Source Type: research
Authors: Mainardi V, Rando K, Valverde M, Olivari D, Castelli J, Rey G, Gerona S Abstract INTRODUCTION AND AIM: Wilson's disease (WD) is an uncommon cause of acute liver failure (ALF). Our aim was to describe clinical features, diagnostic findings, treatments, and outcomes of patients with ALF due to WD. MATERIAL AND METHODS: Retrospective medical record reviews of all patients with ALF due to WD in eight years in Uruguay. RESULTS: WD was the cause of six (15%) of thirty-nine ALF cases. All patients were females, with a mean age of 18 years. Four patients presented with hyperacute liver failure and two with...
Source: Annals of Hepatology - Category: Gastroenterology Tags: Ann Hepatol Source Type: research
Authors: Fonseca F, Kulkarni B, Hastak M, Kumaran V, Varma V, Kapoor S Abstract INTRODUCTION AND AIM: 1. Study of liver explants - Etiologic types of end-stage chronic liver disease (ESCLD) and acute liver failure (ALF) in adults and children. 2. Assessment of donor steatosis and incidental granulomas. 3. Post-transplant liver biopsies. MATERIAL AND METHODS: Specimens of 180 explant hepatectomies, 173 donor wedge and 30 core liver biopsies, and 58 post transplant liver biopsies received in our department from April 2013 to March 2017. RESULTS: 1. Most common causes of ESCLD in adults were: alcohol related (...
Source: Annals of Hepatology - Category: Gastroenterology Tags: Ann Hepatol Source Type: research
Condition:   Wilson's Disease Interventions:   Drug: DMPS;   Drug: Penicillamine;   Drug: DMSA;   Drug: Zinc gluconate Sponsor:   Second Affiliated Hospital, School of Medicine, Zhejiang University Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Wilson ’s disease (WD) is an autosomal recessive disorder caused by mutations in the ATP7B gene. Copper is an essential micronutrient which is incorporated into a variety of proteins and metalloenzymes (cytochrome C oxydase, superoxide dismutase, dopamine-ß hydroxylase, lysil-oxydase, tyrosinase), as we ll as being necessary for the proper growth, development, and function of many organs, including the liver, bone, connective tissue, brain, and heart.1,2
Source: Journal of Hepatology - Category: Gastroenterology Authors: Tags: Hepatology Snapshot Source Type: research
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