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Neurology, ISSN 0028-3878, 11/2018, Volume 91, Issue 21, pp. e1999 - e2009
OBJECTIVETo assess the natural history and treatment effect on survival among patients with transthyretin-associated familial amyloid polyneuropathy (TTR-FAP)... 
LIVER-TRANSPLANTATION | PATISIRAN | THERAPY | EFFICACY | TAFAMIDIS | SAFETY | LONG-TERM SURVIVAL | EPIDEMIOLOGY | CLINICAL NEUROLOGY
Journal Article
Internist, ISSN 0020-9554, 11/2018, Volume 59, Issue 11, pp. 1208 - 1213
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
The New England Journal of Medicine, ISSN 0028-4793, 07/2018, Volume 379, Issue 1, pp. 22 - 31
Journal Article
British Journal of Clinical Pharmacology, ISSN 0306-5251, 06/2015, Volume 79, Issue 6, pp. 918 - 925
Journal Article
Internal Medicine, ISSN 0918-2918, 2017
A 54-year-old man with polycystic liver disease received a domino liver transplantation (DLT) from a patient of hereditary ATTR amyloidosis with the... 
transthyretin | amyloid polyneuropathy | myocardial amyloidosis | tafamidis | domino liver transplantation
Journal Article
JOURNAL OF BIOLOGICAL CHEMISTRY, ISSN 0021-9258, 04/2019, Volume 294, Issue 15, pp. 6130 - 6141
The tetrameric protein transthyretin is a transporter of retinol and thyroxine in blood, cerebrospinal fluid, and the eye, and is secreted by the liver,... 
inhibition mechanism | peptide | POLYNEUROPATHY | amyloidosis | PROTEIN | SAFETY | transthyretin | BIOCHEMISTRY & MOLECULAR BIOLOGY | peptides | inhibition | DIFLUNISAL | POTENT | amyloid | drug discovery | DEPOSITS | seeding | TAFAMIDIS | protein aggregation | aging | AGGREGATION
Journal Article
Ideggyogyaszati Szemle, ISSN 0019-1442, 07/2016, Volume 69, Issue 7-8, pp. 245 - 253
Journal Article
Journal of Neurology, ISSN 0340-5354, 11/2013, Volume 260, Issue 11, pp. 2802 - 2814
Journal Article
Neurology and Clinical Neuroscience, ISSN 2049-4173, 03/2019, Volume 7, Issue 2, pp. 81 - 84
A 71‐year‐old man with a family history of hereditary ATTR ( ATTR m) amyloidosis, began to have sensory disturbance in his extremities at age 65. Although he... 
familial amyloid neuropathy | urodynamics | bladder dysfunction | tafamidis | transthyretin | Bladder
Journal Article
NEUROLOGIA, ISSN 0213-4853, 07/2014, Volume 29, Issue 6, pp. 382 - 384
Journal Article
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, ISSN 0027-8424, 04/2019, Volume 116, Issue 17, pp. 8360 - 8369
Journal Article
Neuroepidemiology, ISSN 0251-5350, 11/2018, Volume 51, Issue 3-4, pp. 177 - 182
Background: Transthyretin-associated familial amyloid polyneuropathy (TTR-FAP) is a rare, hereditary, progressive and neurodegenerative disease. We aimed to... 
Original Paper | PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH | Prevalence | MANAGEMENT | FAP | TAFAMIDIS | Amyloidosis | Transthyretin familial amyloid polyneuropathy | Epidemiology | Portugal | CLINICAL NEUROLOGY | Incidence
Journal Article
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 12/2015, Volume 10, Issue 1, pp. 109 - 109
Journal Article
Neurobiology of Disease, ISSN 0969-9961, 12/2018, Volume 120, pp. 118 - 125
Transthyretin amyloidosis (ATTR) is a fatal disease caused by the systemic aggregation and deposition of transthyretin (TTR), a blood transporter that is... 
Amyloidosis | Peptide inhibitors | Neuropathology | Drosophila | Transthyretin | FIBRIL | LIVER-TRANSPLANTATION | PROTEIN | CARDIOMYOPATHY | NEUROSCIENCES | TAFAMIDIS | FAMILIAL AMYLOIDOTIC POLYNEUROPATHY | MELANOGASTER | PRE-ALBUMIN | Peptides | Analysis | Liver | Transplantation of organs, tissues, etc | Models
Journal Article
Japanese Journal of Geriatrics, ISSN 0300-9173, 2017, Volume 54, Issue 1, pp. 75 - 80
Journal Article