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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Abstract BACKGROUND: Copper (Cu) is a transition metal active in Fenton redox cycling from reduced Cu+ and H2O2, to oxidized Cu2+ and the hydroxyl radical (∙OH) highly reactive oxygen species (ROS). At homeostatic Cu levels, ROS promote cell proliferation, migration, angiogenesis, and wound repair. To limit ROS toxicity, cells use Cu-dependent chaperone proteins, Cu-binding ceruloplasmin, and Cu-modulated enzymes like superoxide dismutases (SOD) like SOD1 and SOD3 to scavenge excess superoxide anion which favours Cu+ reduction, and mitochondrial cytochrome c oxidase, important in aerobic energy production. Becau...
Source: Current Pharmaceutical Design - Category: Drugs & Pharmacology Authors: Tags: Curr Pharm Des Source Type: research
Authors: Kim MK, Lee K, Woo HY, Park H, Ro S, Yoon WT, Kwon MJ PMID: 32539308 [PubMed - in process]
Source: Annals of Laboratory Medicine - Category: Laboratory Medicine Tags: Ann Lab Med Source Type: research
Abstract Caloric restriction mimetics (CRMs) are nontoxic macroautophagy/autophagy enhancers that act through the stimulation of cytoplasmic protein deacetylation reactions. Thus far, three functional classes of CRMs have been described: inhibitors of acetyltransferases (such as spermidine), inhibitors of acetyl coenzyme (AcCoA) synthesis (such as hydroxycitrate) and activators of deacetylases/sirtuins (such as resveratrol). Triethylenetetramine (also called trientine, abbreviated TETA) is a synthetic polyamine with resemblance in its structure to spermidine, a natural polyamine reputed for its pro-autophagic, ant...
Source: Autophagy - Category: Cytology Authors: Tags: Autophagy Source Type: research
Wilson disease (WD) is an Autosomal-Recessive disorder due to mutations of ATP7B gene on chromosome 13q14.3. Inadequate protein function leads to low ceruloplasmin blood levels and copper accumulation in liver, b...
Source: BMC Medical Genetics - Category: Genetics & Stem Cells Authors: Tags: Case report Source Type: research
Condition:   Wilson Disease Intervention:   Drug: Bis-Choline Tetrathiomolybdate Sponsor:   Alexion Pharmaceuticals Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
CONCLUSION: Scheimpflug imaging seems to be a helpful diagnostic tool for detecting and grading the Kayser-Fleischer ring. Corneal thickness in Wilson disease patients with Kayser-Fleischer ring tends to be higher, so that the possible affection in corneal thickness should be kept in mind for clinical evaluation of these patients. PMID: 32515229 [PubMed - as supplied by publisher]
Source: European Journal of Ophthalmology - Category: Opthalmology Authors: Tags: Eur J Ophthalmol Source Type: research
Abstract Copper is an essential element in cells; it can act as either a recipient or a donor of electrons, participating in various reactions. However, an excess of copper ions in cells is detrimental as these copper ions can generate free radicals and increase oxidative stress. In multicellular organisms, copper metabolism involves uptake, distribution, sequestration, and excretion, at both the cellular and systemic levels. Mammalian enterocytes take in bioavailable copper ions from the diet in a Ctr1-dependent manner. After incorporation, cuprous ions are delivered to ATP7A, which pumps Cu+ from enterocytes int...
Source: Pflugers Archiv : European Journal of Physiology - Category: Physiology Authors: Tags: Pflugers Arch Source Type: research
We present a case report of a 32-year-old bodybuilder using myriad nutritional, performance-enhancing, and weight-loss supplements with life-threatening encephalopathy, hepatic failure, rhabdomyolysis, and copper toxicity mimicking Wilson's disease.
Source: The American Journal of Emergency Medicine - Category: Emergency Medicine Authors: Source Type: research
Condition:   Wilson Disease Intervention:   Procedure: Ophthalmological exam Sponsor:   Fondation Ophtalmologique Adolphe de Rothschild Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
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