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.
MeSH terms
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Amyloid Neuropathies, Familial / drug therapy
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Amyloid Neuropathies, Familial / genetics
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Amyloidosis / drug therapy*
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Amyloidosis / genetics
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Amyloidosis / therapy
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Humans
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Immunoglobulin Light Chains / blood
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Immunoglobulin Light Chains / drug effects
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Immunotherapy
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Kidney Transplantation
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Molecular Targeted Therapy*
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Prealbumin / drug effects
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Prealbumin / metabolism*
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Serum Amyloid A Protein / biosynthesis
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Serum Amyloid A Protein / drug effects
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Stem Cell Transplantation
Substances
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Immunoglobulin Light Chains
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Prealbumin
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Serum Amyloid A Protein
Supplementary concepts
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Amyloidosis, Hereditary, Transthyretin-Related