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Aducanumab, sold under the brand name Aduhelm, is a monoclonal antibody designed to treat Alzheimer's disease. It is a monoclonal antibody that targets aggregated forms (plaque) of amyloid beta (Aβ) found in the brains of people with Alzheimer's disease to reduce its buildup. [10] It was developed by Biogen and Eisai. [11] Aducanumab is given via intravenous infusion. [5] Aducanumab was ...
Amyloidosis is a group of diseases in which abnormal proteins, ... liver transplant was the only effective treatment. [5] New therapies include diflunisal, ...
AL amyloidosis is a rare disease; only 1200 to 3200 new cases are reported each year in the United States, and between 500 and 600 in the UK. Two thirds of patients with AL amyloidosis are male and less than 5% of patients are under 40 years of age. [6] [19] [9]
Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. [3] It is a transthyretin-directed antisense oligonucleotide. [3] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca. [4] [5] [6] [7]
Vutrisiran, sold under the brand name Amvuttra, is a medication used for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. [ 7 ] [ 5 ] [ 8 ] It is a double stranded small interfering RNA (siRNA) (also called RNA interference, or RNAi therapeutic) that interferes with the expression of the ...
AA amyloidosis is a form of amyloidosis, a disease characterized by the abnormal deposition of fibers of insoluble protein in the extracellular space of various tissues and organs. In AA amyloidosis, the deposited protein is serum amyloid A protein (SAA), an acute-phase protein which is normally soluble and whose plasma concentration is highest ...
These therapies target hereditary angioedema (HAE) and transthyretin (ATTR) amyloidosis, respectively. Intellia prioritized NTLA-2002 and nex-z while discontinuing the NTLA-3001 program and other ...
Tafamidis was approved in the European Union in 2011 for the treatment of transthyretin amyloidosis with polyneuropathy, and in Japan in 2013. [6] [17] In the United States, it was rejected for the treatment of transthyretin amyloidosis with polyneuropathy because the Food and Drug Administration saw insufficient evidence for its efficacy. [20] [7]
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