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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
JAMA Neurology, ISSN 2168-6149, 04/2015, Volume 72, Issue 4, pp. 396 - 404
Journal Article
Human Mutation, ISSN 1059-7794, 09/2015, Volume 36, Issue 9, pp. 881 - 893
Journal Article
Developmental Medicine & Child Neurology, ISSN 0012-1622, 06/2011, Volume 53, Issue 6, pp. 565 - 568
Journal Article
Neuromuscular Disorders, ISSN 0960-8966, 2008, Volume 18, Issue 8, pp. 626 - 632
Journal Article
British Columbia Medical Journal, ISSN 0007-0556, 05/2011, Volume 53, Issue 4, pp. 183 - 187
Journal Article
British Columbia Medical Journal, ISSN 0007-0556, 05/2011, Volume 53, Issue 4, pp. 177 - 182
Journal Article
JIMD Reports, ISSN 2192-8304, 2014, Volume 12, pp. 17 - 23
The pathophysiology of hypertension in patients with mitochondrial diseases is different from that of the general population. Growing evidence exists linking... 
Common clinical manifestation | Canadian health measure survey | Skeletal myopathy | Mitochondrial haplogroups | Mitochondrial tRNA
Journal Article
Journal Article
by Wolfe, Gil I and Wolfe, Gil and Kaminski, Henry and Kaminski, Henry J and Aban, Inmaculada B and Aban, Inmaculada and Minisman, Greg and Kuo, Hui-Chien and Marx, Alexander and Ströbel, Philipp and Mazia, Claudio and Oger, Joel and Cea, Gabriel and Cea, J Gabriel and Heckmann, Jeannine M and Heckmann, Jeannine and Evoli, Amelia and Nix, Wilfred and Ciafaloni, Emma and Antonini, Giovanni and Witoonpanich, Rawiphan and King, John and King, John O and Beydoun, Said and Beydoun, Said R and Chalk, Colin and Chalk, Colin H and Barboi, Alexandru C and Barboi, Alexandru and Amato, Anthony A and Amato, Anthony and Shaibani, Aziz I and Shaibani, Aziz and Katirji, Bashar and Lecky, Bryan R F and Lecky, Bryan and Buckley, Camilla and Vincent, Angela and Dias-Tosta, Elza and Yoshikawa, Hiroaki and Waddington-Cruz, Márcia and Pulley, Michael T and Pulley, Michael and Rivner, Michael H and Rivner, Michael and Kostera-Pruszczyk, Anne and Kostera-Pruszczyk, Anna and Pascuzzi, Robert M and Pascuzzi, Robert and Jackson, Carlayne E and Jackson, Carlayne and Verschuuren, Jan and Verschuuren, Jan J G M and Massey, Janice M and Massey, Janice and Kissel, John T and Kissel, John and Werneck, Lineu C and Werneck, Lineu and Benatar, Michael and Barohn, Richard and Barohn, Richard J and Tandan, Rup and Mozaffar, Tahseen and Silvestri, Nicholas J and Conwit, Robin and Sonett, Joshua R and Sonett, Joshua and Jaretzki, Alfred and Newsom-Davis, John and Cutter, Gary and Cutter, Gary R and Feese, Michelle and Saluto, Valeria and Rosenberg, Moises and Alvarez, Valeria and Rey, Lisa and Butzkueven, Helmut and Goldblatt, John and Carey, John and Pollard, John and Reddel, Stephen and Handel, Nicholas and McCaughan, Brian and Pallot, Linda and Novis, Ricardo and Boasquevisque, Carlos and Morato-Fernandez, Rubens and Ximenes, Manoel and Scola, Rosana and Soltoski, Paulo and Moore, Fraser and Mulder, David and Wadup, Lisa and Mezei, Michele and Evans, Kenneth and Jiwa, Theresa and Schaffar, Anne and White, Chris and Toth, Cory and ... and MGTX Study Grp and MGTX Study Group
The Lancet Neurology, ISSN 1474-4422, 03/2019, Volume 18, Issue 3, pp. 259 - 268
The Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone (MGTX) showed that thymectomy combined with prednisone was superior to... 
CLINICAL NEUROLOGY | QUALITY-OF-LIFE | Care and treatment | Prednisone | Corticosteroids | Myasthenia gravis | Analysis | Steroids | Corticoids | Immunoglobulins | Stroke | Patients | Studies | Neurological diseases | Thymectomy | Motivation | Neuromuscular junctions | Myasthenia | Acetylcholine | EMG | Long-term effects
Journal Article