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Emerging Treatments for Amyloidosis

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source: Kidney International

year: 2015

authors: Sayed RH, Hawkins PN, Lachmann HJ

summary/abstract:

Amyloidosis results from protein misfolding, and ongoing amyloid deposition can ultimately lead to organ failure and death. Historically, this is a group of diseases with limited treatment options and frequently poor prognosis. However, there are now ‘targeted’ therapeutics emerging in the form of stabilizers of the precursor protein, inhibitors of fibrillogenesis, fibril disruptors, and blockers of protein translation, transcription, and immunotherapy. We review many of these approaches that are currently being assessed in clinical trials.

organization: UCL Medical School, UK

DOI: 10.1038/ki.2014.368

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