title: Emerging treatments for amyloidosis.
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.
authors.affiliation: 1] National Amyloidosis Centre, UCL Medical School, Royal Free Hospital, London, UK [2] UCL Centre for Nephrology, UCL Medical School, Royal Free Hospital, London, UK.
authors.forename: Rabya H
authors.initials: RH
authors.lastname: Sayed
authors.affiliation: National Amyloidosis Centre, UCL Medical School, Royal Free Hospital, London, UK.
authors.forename: Philip N
authors.initials: PN
authors.lastname: Hawkins
authors.affiliation: 1] National Amyloidosis Centre, UCL Medical School, Royal Free Hospital, London, UK [2] UCL Centre for Nephrology, UCL Medical School, Royal Free Hospital, London, UK.
authors.forename: Helen J
authors.initials: HJ
authors.lastname: Lachmann
mesh_terms: D028227:Amyloid Neuropathies, Familial / Q000188:drug therapy / Q000235:genetics; D000686:Amyloidosis / Q000188:drug therapy* / Q000235:genetics / Q000628:therapy; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000097:blood / Q000187:drug effects; D007167:Immunotherapy; D016030:Kidney Transplantation; D058990:Molecular Targeted Therapy; D011228:Prealbumin / Q000187:drug effects / Q000378:metabolism*; D000685:Serum Amyloid A Protein / Q000096:biosynthesis / Q000187:drug effects; D033581:Stem Cell Transplantation
chemical_list: D007147:Immunoglobulin Light Chains; D011228:Prealbumin; D000685:Serum Amyloid A Protein