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The New England Journal of Medicine, ISSN 0028-4793, 09/2012, Volume 367, Issue 12, pp. 1098 - 1107
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 09/2012, Volume 367, Issue 12, pp. 1087 - 1097
Journal Article
Journal Article
Journal of Clinical Pharmacology, ISSN 0091-2700, 07/2019
ABSTRACTHereditary transthyretin-mediated (hATTR) amyloidosis is an inherited, rapidly progressive, life-threatening disease caused by deposition of abnormal... 
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 07/2018, Volume 379, Issue 1, pp. 11 - 21
Hereditary transthyretin amyloidosis is caused by the deposition of misfolded transthyretin proteins in peripheral nerves and other tissues. This phase 3 trial... 
MEDICINE, GENERAL & INTERNAL | POLYNEUROPATHY | EFFICACY | TAFAMIDIS | SAFETY | LATE-ONSET | NATURAL-HISTORY | PROGRESSION | Prealbumin - genetics | Humans | Middle Aged | Male | Amyloid Neuropathies, Familial - therapy | Infusions, Intravenous - adverse effects | Aged, 80 and over | Adult | Female | Prealbumin - analysis | Severity of Illness Index | Double-Blind Method | Amyloid Neuropathies, Familial - complications | RNA, Small Interfering - adverse effects | Polyneuropathies - therapy | Disease Progression | Administration, Intravenous | Polyneuropathies - etiology | RNA, Small Interfering - therapeutic use | Least-Squares Analysis | Quality of Life | Aged | RNAi Therapeutics | Amyloid Neuropathies, Familial - blood | Edema - chemically induced | Walk Test | Gait Disorders, Neurologic - etiology | Care and treatment | Amyloidosis | Genetic aspects | Intravenous administration | Gait | Cardiomyopathy | RNA-mediated interference | Liver | Diabetes mellitus | Body weight | Transthyretin | Diabetic neuropathy | Neuropathy | Gene expression | Polyneuropathy | Quality of life | Medicine | Neurology | Hospitals | Nutritional status | Pharmaceuticals | Index Medicus | Abridged Index Medicus | Assaigs clínics | Clinical trials | RNA | Amiloïdosi | Clinical Medicine | Neurologi | Medical and Health Sciences | Klinisk medicin | Medicin och hälsovetenskap
Journal Article
New England Journal of Medicine, ISSN 0028-4793, 2013, Volume 369, Issue 11, p. 1082
Journal Article
Multiple Sclerosis Journal, ISSN 1352-4585, 2/2017, Volume 23, Issue 2, pp. 253 - 265
Journal Article
Journal Article