TY - JOUR AU - Ando, Y. AU - Nakamura, M. AU - Araki, S. PY - 2005 DA - 2005// TI - Transthyretin-related familial amyloidotic polyneuropathy JO - Arch Neurol VL - 62 UR - https://doi.org/10.1001/archneur.62.7.1057 DO - 10.1001/archneur.62.7.1057 ID - Ando2005 ER - TY - JOUR AU - Bolte, F. J. AU - Schmidt, H. H. AU - Becker, T. AU - Braun, F. AU - Pascher, A. AU - Klempnauer, J. PY - 2013 DA - 2013// TI - Evaluation of domino liver transplantations in Germany JO - Transpl Int VL - 26 UR - https://doi.org/10.1111/tri.12110 DO - 10.1111/tri.12110 ID - Bolte2013 ER - TY - JOUR AU - Schmidt, H. AU - Cruz, M. W. AU - Botteman, M. F. AU - Carter, J. A. AU - Chopra, A. AU - Stewart, M. PY - 2017 DA - 2017// TI - Global epidemiology of transthyretin hereditary amyloid polyneuropathy: a systematic review JO - Amyloid VL - 24 UR - https://doi.org/10.1080/13506129.2017.1292903 DO - 10.1080/13506129.2017.1292903 ID - Schmidt2017 ER - TY - JOUR AU - Schmidt, H. H. AU - Waddington-Cruz, M. AU - Botteman, M. F. AU - Carter, J. A. AU - Chopra, A. S. AU - Hopps, M. PY - 2018 DA - 2018// TI - Estimating the global prevalence of transthyretin familial amyloid polyneuropathy JO - Muscle Nerve VL - 57 UR - https://doi.org/10.1002/mus.26034 DO - 10.1002/mus.26034 ID - Schmidt2018 ER - TY - JOUR AU - Waddington-Cruz, M. AU - Schmidt, H. AU - Botteman, M. F. AU - Carter, J. A. AU - Stewart, M. AU - Hopps, M. PY - 2019 DA - 2019// TI - Epidemiological and clinical characteristics of symptomatic hereditary transthyretin amyloid polyneuropathy: a global case series JO - Orphanet J Rare Dis VL - 14 UR - https://doi.org/10.1186/s13023-019-1000-1 DO - 10.1186/s13023-019-1000-1 ID - Waddington-Cruz2019 ER - TY - JOUR AU - Benson, M. D. AU - Kincaid, J. C. PY - 2007 DA - 2007// TI - The molecular biology and clinical features of amyloid neuropathy JO - Muscle Nerve VL - 36 UR - https://doi.org/10.1002/mus.20821 DO - 10.1002/mus.20821 ID - Benson2007 ER - TY - JOUR AU - Conceicao, I. AU - Gonzalez-Duarte, A. AU - Obici, L. AU - Schmidt, H. H. AU - Simoneau, D. AU - Ong, M. L. PY - 2016 DA - 2016// TI - "Red-flag" symptom clusters in transthyretin familial amyloid polyneuropathy JO - J Peripher Nerv Syst VL - 21 UR - https://doi.org/10.1111/jns.12153 DO - 10.1111/jns.12153 ID - Conceicao2016 ER - TY - STD TI - Gonzalez-Duarte A, Valdes-Ferrer SI, Cantu-Brito C. Characteristics and natural history of autonomic involvement in hereditary ATTR amyloidosis: a systematic review. Clin Auton Res. 2019. ID - ref8 ER - TY - JOUR AU - Gillmore, J. D. AU - Maurer, M. S. AU - Falk, R. H. AU - Merlini, G. AU - Damy, T. AU - Dispenzieri, A. PY - 2016 DA - 2016// TI - Nonbiopsy diagnosis of cardiac transthyretin amyloidosis JO - Circulation VL - 133 UR - https://doi.org/10.1161/CIRCULATIONAHA.116.021612 DO - 10.1161/CIRCULATIONAHA.116.021612 ID - Gillmore2016 ER - TY - JOUR AU - Adams, D. AU - Samuel, D. AU - Goulon-Goeau, C. AU - Nakazato, M. AU - Costa, P. M. AU - Feray, C. PY - 2000 DA - 2000// TI - The course and prognostic factors of familial amyloid polyneuropathy after liver transplantation JO - Brain VL - 123 UR - https://doi.org/10.1093/brain/123.7.1495 DO - 10.1093/brain/123.7.1495 ID - Adams2000 ER - TY - JOUR AU - Laugsand, E. A. AU - Sprangers, M. A. AU - Bjordal, K. AU - Skorpen, F. AU - Kaasa, S. AU - Klepstad, P. PY - 2010 DA - 2010// TI - Health care providers underestimate symptom intensities of cancer patients: a multicenter European study JO - Health Qual. Life Outcomes VL - 8 UR - https://doi.org/10.1186/1477-7525-8-104 DO - 10.1186/1477-7525-8-104 ID - Laugsand2010 ER - TY - JOUR AU - Conceição, I. AU - Coelho, T. AU - Rapezzi, C. AU - Parman, Y. AU - Obici, L. AU - Galán, L. PY - 2019 DA - 2019// TI - Assessment of patients with hereditary transthyretin amyloidosis – understanding the impact of management and disease progression JO - Amyloid VL - 26 UR - https://doi.org/10.1080/13506129.2019.1627312 DO - 10.1080/13506129.2019.1627312 ID - Conceição2019 ER - TY - JOUR AU - Ando, Y. AU - Coelho, T. AU - Berk, J. L. AU - Cruz, M. W. AU - Ericzon, B. G. AU - Ikeda, S. PY - 2013 DA - 2013// TI - Guideline of transthyretin-related hereditary amyloidosis for clinicians JO - Orphanet J Rare Dis VL - 8 UR - https://doi.org/10.1186/1750-1172-8-31 DO - 10.1186/1750-1172-8-31 ID - Ando2013 ER - TY - JOUR AU - Dyck, P. J. AU - Kincaid, J. C. AU - Dyck, P. J. B. AU - Chaudhry, V. AU - Goyal, N. A. AU - Alves, C. PY - 2017 DA - 2017// TI - Assessing mNIS+7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial JO - Muscle Nerve VL - 56 UR - https://doi.org/10.1002/mus.25563 DO - 10.1002/mus.25563 ID - Dyck2017 ER - TY - JOUR AU - Pinto, M. V. AU - Barreira, A. A. AU - Bulle, A. S. AU - Freitas, M. R. G. AU - Franca, M. C. AU - Gondim, F. A. A. PY - 2018 DA - 2018// TI - Brazilian consensus for diagnosis, management and treatment of transthyretin familial amyloid polyneuropathy JO - Arq Neuropsiquiatr VL - 76 UR - https://doi.org/10.1590/0004-282x20180094 DO - 10.1590/0004-282x20180094 ID - Pinto2018 ER - TY - JOUR AU - Ong, W. L. AU - Schouwenburg, M. G. AU - Bommel, A. C. M. AU - Stowell, C. AU - Allison, K. H. AU - Benn, K. E. PY - 2017 DA - 2017// TI - A standard set of value-based patient-centered outcomes for breast cancer: the international consortium for health outcomes measurement (ICHOM) initiative JO - JAMA Oncol VL - 3 UR - https://doi.org/10.1001/jamaoncol.2016.4851 DO - 10.1001/jamaoncol.2016.4851 ID - Ong2017 ER - TY - JOUR AU - Morel, T. AU - Cano, S. J. PY - 2017 DA - 2017// TI - Measuring what matters to rare disease patients - reflections on the work by the IRDiRC taskforce on patient-centered outcome measures JO - Orphanet J Rare Dis VL - 12 UR - https://doi.org/10.1186/s13023-017-0718-x DO - 10.1186/s13023-017-0718-x ID - Morel2017 ER - TY - JOUR AU - Benson, M. D. AU - Waddington-Cruz, M. AU - Berk, J. L. AU - Polydefkis, M. AU - Dyck, P. J. AU - Wang, A. K. PY - 2018 DA - 2018// TI - Inotersen treatment for patients with hereditary transthyretin amyloidosis JO - N Engl J Med VL - 379 UR - https://doi.org/10.1056/NEJMoa1716793 DO - 10.1056/NEJMoa1716793 ID - Benson2018 ER - TY - JOUR AU - Adams, D. AU - Gonzalez-Duarte, A. AU - O'Riordan, W. D. AU - Yang, C. C. AU - Ueda, M. AU - Kristen, A. V. PY - 2018 DA - 2018// TI - Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis JO - N Engl J Med VL - 379 UR - https://doi.org/10.1056/NEJMoa1716153 DO - 10.1056/NEJMoa1716153 ID - Adams2018 ER - TY - JOUR AU - Hays, R. D. AU - Spritzer, K. L. AU - Thompson, W. W. AU - Cella, D. U. S. PY - 2015 DA - 2015// TI - General population estimate for "excellent" to "poor" self-rated health item JO - J Gen Intern Med VL - 30 UR - https://doi.org/10.1007/s11606-015-3290-x DO - 10.1007/s11606-015-3290-x ID - Hays2015 ER - TY - JOUR AU - Kilo, C. M. AU - Kabcenell, A. AU - Berwick, D. M. PY - 1998 DA - 1998// TI - Beyond survival: toward continuous improvement in medical care JO - New Horiz VL - 6 ID - Kilo1998 ER - TY - STD TI - Licqurish SM, Cook OY, Pattuwage LP, Saunders C, Jefford M, Koczwara B, et al. Tools to facilitate communication during physician-patient consultations in cancer care: an overview of systematic reviews. CA Cancer J Clin. 2019. ID - ref22 ER - TY - JOUR AU - Ustun, T. B. AU - Chatterji, S. AU - Kostanjsek, N. AU - Rehm, J. AU - Kennedy, C. AU - Epping-Jordan, J. PY - 2010 DA - 2010// TI - Developing the World Health Organization disability assessment schedule 2.0 JO - Bull World Health Organ. VL - 88 UR - https://doi.org/10.2471/BLT.09.067231 DO - 10.2471/BLT.09.067231 ID - Ustun2010 ER -