The European Medicines Authority’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for a new drug to treat hereditary angioedema (HAE).
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1. In this randomized controlled trial, 80 mg of olezarsen, but not 50 mg, significantly reduced triglyceride levels compared to placebo after six months in familial hyperchylomicronemia syndrome (FCS ...
AGT (angiotensinogen) serves as the exclusive substrate in the renin-angiotensin system. Deleting AGT, through suppressing its synthesis in hepatocytes, improves cardiovascular functions attributed to ...
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Hereditary transthyretin-related (hATTR) amyloidosis is a rare disorder that is fatal if left untreated. The disease is caused by mutations in the transthyretin (TTR) gene and is inherited in an ...
In recent years, mesoporous silica particles have been revealed as promising drug delivery systems combining high drug loading capacity, excellent biocompatibility, and easy and affordable synthetic ...
Hereditary angioedema is characterized by recurrent and unpredictable swellings that are disabling and potentially fatal. Selective inhibition of plasma prekallikrein production by antisense ...
Glycosylation is a critical quality attribute of monoclonal antibody (mAb) therapeutics. Hydrophilic interaction liquid chromatography-mass spectrometry (HILIC-MS) is an invaluable technology for the ...
Nonalcoholic steatohepatitis (NASH) is a progressive form of nonalcoholic fatty liver disease that can lead to irreversible liver cirrhosis and cancer. Early diagnosis of NASH is vital to detect ...
While type IV pili (Tfp) are one of the most widespread adhesive factors found in prokaryotes, little is known about their cellular targets. Obtaining a better understanding of the molecular basis of ...
Lipoprotein(a) levels are genetically determined and, when elevated, are a risk factor for cardiovascular disease and aortic stenosis. There are no approved pharmacologic therapies to lower ...