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Assessment of immunogenicity from the pegunigalsidase alfa clinical trial program: Integrated analysis of de novo and treatment-boosted anti-drug antibodies Bernat, John; Hughes, Derralynn; Linhart, Ales; Hopkin, Robert J.; Tondel, Camilla; Warnock, David G.; Rup, Bonita; Koulinska, Irene; Rocco, Rossana; Alon, Sari; Chertkoff, Raul; Almon, Einat Brill; Sakov, Anat; Wallace, Eric 分享 收藏
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study Wallace, Eric L.; Goker-Alpan, Ozlem; Wilcox, William R.; Holida, Myrl; Bernat, John; Longo, Nicola; Linhart, Ales; Hughes, Derralynn A.; Hopkin, Robert J.; Tondel, Camilla; Langeveld, Mirjam; Giraldo, Pilar; Pisani, Antonio; Germain, Dominique Paul; Mehta, Ankit; Deegan, Patrick B.; Molnar, Maria Judit; Ortiz, Damara; Jovanovic, Ana; Muriello, Michael; Barshop, Bruce A.; Kimonis, Virginia; Vujkovac, Bojan; Nowak, Albina; Geberhiwot, Tarekegn; Kantola, Ilkka; Knoll, Jasmine; Waldek, Stephen; Nedd, Khan; Karaa, Amel; Brill-Almon, Einat; Alon, Sari; Chertkoff, Raul; Rocco, Rossana; Sakov, Anat; Warnock, David G. 分享 收藏
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First results of a head-to-head trial of pegunigalsidase alfa vs. agalsidase beta in Fabry disease: 2 year results of the phase 3 randomized, double-blind, BALANCE study Wallace, Eric; Goker-Alpan, Wilcox; Holida, Bernat; Longo, N.; Hughes, D.; Giraldo, P.; Molnar, M. J.; Ortiz, Hopkin; Tondel, C.; Linhart, A.; Deegan, Jovanovic; Muriello, M.; Barshop, Kimonis; Vujkovac, B.; Nowak, A.; Geberhiwot, T.; Pisani, A.; Germain, D. P.; Kantola, I.; Knoll, J.; Mehta, Ankit; Waldek, Stephen; Chertkoff, Raul; Almon, Einat; Alon, Sari; Rocco, Rossana; Warnock, David G. 分享 收藏
Estimation of arrhythmia risk in patients with Fabry disease using a machine learning model Aguiar, Patricio; Biondetti, Giorgio Pietro; Warnock, David G.; Kallish, Staci; Nelson, Matthew W.; Giuliano, Joseph; Zabinski, Joseph W.; Boussios, Costas; Curhan, Gary; Bandaria, Jigar N.; Gliklich, Richard E.; Jefferies, John L. 分享 收藏
Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: Twoyear interim results from the ongoing phase 3 BRIGHT51 openlabel extension study Bernat, John; Holida, Myrl D.; Longo, Nicola; Goker-Alpan, Ozlem; Wallace, Eric; Deegan, Patrick B.; Tondel, Camilla; Eyskens, Francois J.; Feldt-Rasmussen, Ulla; Hughes, Derralynn; Pisani, Antonio; Rocco, Rossana; Almon, Einat Brill; Alon, Sari; Chertkoff, Raul; Warnock, David G.; Waldek, Stephen; Wilcox, William R. 分享 收藏
Safety and efficacy of pegunigalsidase alfa, every 4 weeks, in Fabry disease: Results from the phase 3, open-label, BRIGHT study Bernat, John; Holida, Myrl; Longo, Nicola; Goker-Alpan, Ozlem; Wallace, Eric; Deegan, Patrick; Nedd, Khan; Tondel, Camilla; Eyskens, Francois; Feldt-Rasmussen, Ulla; Pisani, Antonio; Rocco, Rossana; Almon, Einat; Alon, Sari; Chertkoff, Raul; Warnock, David; Waldek, Stephen; Wilcox, William 分享 收藏
A new approach to identifying patients with elevated risk for Fabry disease using a machine learning algorithm Jefferies, John L.; Spencer, Alison K.; Lau, Heather A.; Nelson, Matthew W.; Giuliano, Joseph D.; Zabinski, Joseph W.; Boussios, Costas; Curhan, Gary; Gliklich, Richard E.; Warnock, David G. 分享 收藏
Standardising clinical outcomes measures for adult clinical trials in Fabry disease: A global Delphi consensus Moreno-Martinez, D.; Aguiar, P.; Auray-Blais, C.; Beck, M.; Bichet, D. G.; Burlina, A.; Cole, D.; Elliott, P.; Feldt-Rasmussen, U.; Feriozzi, S.; Fletcher, J.; Giugliani, R.; Jovanovic, A.; Kampmann, C.; Langeveld, M.; Lidove, O.; Linhart, A.; Mauer, M.; Moon, J. C.; Muir, A.; Nowak, A.; Oliveira, J. P.; Ortiz, A.; Pintos-Morell, G.; Politei, J.; Rozenfeld, P.; Schiffmann, R.; Svarstad, E.; Talbot, A. S.; Thomas, M.; Tondel, C.; Warnock, D.; West, M. L.; Hughes, D. A. 分享 收藏
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Use of a rare disease registry for establishing phenotypic classification of previously unassigned GLA variants: a consensus classification system by a multispecialty Fabry disease genotype-phenotype workgroup 使用罕见疾病注册表建立以前未分配的GLA变体的表型分类: 多专业法布里病基因型-表型工作组的共识分类系统 Germain, Dominique P.; Oliveira, Joao Paulo; Bichet, Daniel G.; Yoo, Han-Wook; Hopkin, Robert J.; Lemay, Roberta; Politei, Juan; Wanner, Christoph; Wilcox, William R.; Warnock, David G. 分享 收藏
Rationale and design of the MODIFY study: A phase 3 multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease Wanner, Christoph; Hughes, Derralynn; Kimonis, Virginia; Politei, Juan; Uceyler, Nurcan; Warnock, David G.; Cornelisse, Peter; Frey, Aline 分享 收藏
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Rapid, proteomic urine assay for monitoring progressive organ disease in Fabry disease Doykov, Ivan D.; Heywood, Wendy E.; Nikolaenko, Valeria; Spiewak, Justyna; Hallqvist, Jenny; Clayton, Peter Theodore; Mills, Philippa; Warnock, David G.; Nowak, Albina; Mills, Kevin 分享 收藏
Analysis of the baseline characteristics of Fabry disease patients screened for the pegunigalsidase alfa phase III BALANCE study Warnock, David; Eric, Wallace; Schiffmann, Raphael; Holida, Myrl; Goker-Alpan, Ozlem; Hopkin, Robert; Giraldo, Pilar; Khan, Nedd; Vujkovac, Bojan; Wilcox, William; Linhart, Ales; Tondel, Camilla; Barshop, Bruce A.; Karaa, Amel; Molnar, Maria Judit; Deegan, Patrick; Vockley, Jerry; Rhead, William; Kimonis, Virginia; Chertkoff, Raul; Longo, Nicola 分享 收藏
Urinary Tubular Injury Biomarkers Are Associated With ESRD and Death in the REGARDS Study Dubin, Ruth F.; Judd, Suzanne; Scherzer, Rebecca; Shlipak, Michael; Warnock, David G.; Cushman, Mary; Sarnak, Mark; Parikh, Chirag; Bennett, Michael; Powe, Neil; Peralta, Carmen A. 分享 收藏
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A survivor analysis for major clinical events in heterozygous female patients with Fabry disease using group consensus phenotype classifications from hemizygous male patients Hopkin, Robert J.; Germain, Dominique P.; Bichet, Daniel G.; Gruskin, Daniel J.; Lemay, Roberta M.; Oliveira, Joao P.; Politei, Juan M.; Wanner, Christoph; Wilcox, William R.; Yoo, Han-Wook; Warnock, David G. 分享 收藏
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