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Emerging treatments for amyloidosis
Author(s) -
Rabya Sayed,
Philip N. Hawkins,
Helen J. Lachmann
Publication year - 2014
Publication title -
kidney international
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.499
H-Index - 276
eISSN - 1523-1755
pISSN - 0085-2538
DOI - 10.1038/ki.2014.368
Subject(s) - amyloidosis , medicine , fibrillogenesis , al amyloidosis , amyloid fibril , amyloid (mycology) , clinical trial , immunology , disease , pathology , immunoglobulin light chain , antibody , chemistry , fibril , amyloid β , biochemistry
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.

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