1.
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15 p, 1.1 MB |
Do clinical guidelines facilitate or impede drivers of treatment in Fabry disease?
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Hughes, Derralynn A. (University College London) ;
Aguiar, Patrício (Lisbon University) ;
Lidove, Olivier (Croix Saint Simon Hospital, Paris) ;
Nicholls, Kathleen (University of Melbourne) ;
Nowak, Albina (University Hospital Zurich (Suïssa)) ;
Thomas, Mark (Cincinnati Children's Hospital Medical Center (CCHMC). Center for Fetal and Placental Research) ;
Torra Balcells, Roser (Institut d'Investigació Biomèdica Sant Pau) ;
Vujkovac, Bojan (General Hospital Slovenj Gradec) ;
West, Michael L. (Dalhousie University) ;
Feriozzi, Sandro (Belcolle Hospital) ;
Universitat Autònoma de Barcelona
Variable disease progression confounds accurate prognosis in Fabry disease. Evidence supports the long-term benefit of early intervention with disease-specific therapy, but current guidelines recommend treatment initiation based on signs that may present too late to avoid irreversible organ damage. [...]
2022 - 10.1186/s13023-022-02181-4
Orphanet Journal of Rare Diseases, Vol. 17 (february 2022)
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2.
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12 p, 1.2 MB |
Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation : findings from the opinion-based PREDICT-FD modified Delphi consensus initiative
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Hughes, Derralynn A. (University College London) ;
Aguiar, Patrício (University of Lisbon) ;
Deegan, Patrick B. (University of Cambridge) ;
Ezgu, Fatih (Gazi University) ;
Frustaci, Andrea (University of Rome La Sapienza) ;
Lidove, Olivier (Croix Saint Simon Hospital) ;
Linhart, Aleš (Charles University. Faculty of Medicine in Hradec Králové) ;
Lubanda, Jean-Claude (Charles University. Faculty of Medicine in Hradec Králové) ;
Moon, James C. (Barts Heart Centre) ;
Nicholls, Kathleen (The University of Melbourne) ;
Niu, Dau-Ming (National Yang-Ming University) ;
Nowak, Albina (University Hospital Zurich (Suïssa)) ;
Ramaswami, Uma (Royal Free Hospital) ;
Reisin, Ricardo (British Hospital of Buenos Aires) ;
Rozenfeld, Paula (Instituto de Estudios Inmunológicos y Fisiopatológicos, UNLP - CONICET, La Plata, Argentina) ;
Schiffmann, Raphael (Baylor Research Institute) ;
Svarstad, Einar (Haukeland University Hospital (Bergen, Noruega)) ;
Thomas, Mark (Cincinnati Children's Hospital Medical Center) ;
Torra Balcells, Roser (Institut d'Investigació Biomèdica Sant Pau) ;
Vujkovac, Bojan (General Hospital Slovenj Gradec) ;
Warnock, David G. (University of Alabama at Birmingham) ;
West, Michael L. (Dalhousie University) ;
Johnson, Jack (Fabry International Network) ;
Rolfe, Mark J. (Oxford Pharmagenesis) ;
Feriozzi, Sandro (Belcolle Hospital) ;
Universitat Autònoma de Barcelona
The PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease (PREDICT-FD) initiative aimed to reach consensus among a panel of global experts on early indicators of disease progression that may justify FD-specific treatment initiation. [...]
2020 - 10.1136/bmjopen-2019-035182
BMJ open, Vol. 10 (october 2020)
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3.
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11 p, 6.0 MB |
Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy : A 10-year fabry outcome survey (FOS) analysis
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Ramaswami, Uma (Royal Free London NHS Foundation Trust. Lysosomal Disorders Unit. Institute of Immunity and Transplantation) ;
Beck, Michael (Centre for Paediatric and Adolescent Medicine. University Medical Centre. University of Mainz) ;
Hughes, D. (Royal Free London NHS Foundation Trust. Lysosomal Disorders Unit. Institute of Immunity and Transplantation) ;
Kampmann, C. (Centre for Paediatric and Adolescent Medicine. University Medical Centre. University of Mainz) ;
Botha, J. (Department of Biostatistics and Programming. Takeda) ;
Pintos-Morell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ;
West, M. L. (Department of Medicine. Dalhousie University) ;
Niu, D. M. (Department of Paediatrics. Taipei Veterans General Hospital) ;
Nicholls, K. (Royal Melbourne Hospital (Melbourne, Austràlia)) ;
Giugliani, R. (Medical Genetics Service HCPA. Department of Genetics. UFRGS. INAGEMP) ;
Universitat Autònoma de Barcelona
Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials. [...]
2019 - 10.2147/DDDT.S207856
Drug Design, Development and Therapy, Vol. 13 (2019) , p. 3705-3715
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4.
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30 p, 610.3 KB |
Considering weed management as a social dilemma bridges individual and collective interests
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Bagavathiannan, Muthukumar V. (Texas A&M University. Department of Soil and Crop Sciences) ;
Graham, Sonia (Universitat Autònoma de Barcelona. Institut de Ciència i Tecnologia Ambientals) ;
Ma, Zhao (Purdue University. Department of Forestry and Natural Resources) ;
Barney, Jacob N. (School of Plant and Environmental Sciences) ;
Coutts, Shaun R. (University of Sheffield) ;
Caicedo, Ana L. (University of Massachusetts. Biology Department) ;
De Clerck-Floate, Rosemarie (Agriculture and Agri-Food Canada) ;
West, Natalie M. (United States Department of Agriculture - Agricultural Research Service) ;
Blank, Lior (Volcani Center) ;
Metcalf, Alexander L. (University of Montana) ;
Lacoste, Myrtille (The University of Western Australia) ;
Moreno, Carlo (The College of Wooster) ;
Evans, Jeffrey A. (United States Department of Agriculture - Agricultural Research Service) ;
Burke, Ian (Washington State University. Department of Crop and Soil Sciences) ;
Beckie, Hugh (Agriculture and Agri-Food Canada)
Weeds pose severe threats to agricultural and natural landscapes worldwide. One major reason for the failure to effectively manage weeds at landscape scales is that current Best Management Practice guidelines, and research on how to improve such guidelines, focus too narrowly on property-level management decisions. [...]
2019 - 10.1038/s41477-019-0395-y
Nature plants, Vol. 5 (April 2019) , p. 343-351
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5.
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5 p, 246.4 KB |
Long-term outcomes with agalsidase alfa enzyme replacement therapy : analysis using deconstructed composite events
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Beck, Michael (University Medical Center, Mainz, Germany) ;
Hughes, Derralynn A (University College London) ;
Kampmann, Christoph (University Medical Center, Mainz, Germany) ;
Pintos-Morell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ;
Ramaswami, Uma (University College London) ;
West, Michael L. (Dalhousie University, Halifax, Canada) ;
Giugliani, Roberto (Universidade Federal do Rio Grande do Sul, Brazil) ;
Universitat Autònoma de Barcelona
This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. [...]
2017 - 10.1016/j.ymgmr.2017.10.008
Molecular Genetics and Metabolism Reports, Vol. 14 (november 2017) , p. 31-35
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6.
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7 p, 412.7 KB |
Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease : A Fabry Outcome Survey analysis
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Beck, Michael (University Medical Center, University of Mainz, Department of Paediatrics) ;
Hughes, Derralynn A (Royal Free London NHS Foundation Trust, University College of London) ;
Kampmann, Christoph (University Medical Center, University of Mainz, Department of Paediatrics) ;
Larroque, Sylvain (Shire, Zug) ;
Mehta, Atul (Royal Free London NHS Foundation Trust, University College of London) ;
Pintos-Morellell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ;
Ramaswami, Uma (Royal Free London NHS Foundation Trust, University College of London) ;
West, Michael (Department of Medicine, Dalhousie University) ;
Wijatyk, Anna (Shire) ;
Giugliani, Roberto (Medical Genetics Service HCPA/Dep Genet UFRGS and INAGEMP, Porto Alegre) ;
Universitat Autònoma de Barcelona
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease. [...]
2015 - 10.1016/j.ymgmr.2015.02.002
Molecular Genetics and Metabolism Reports, Vol. 3 (march 2015) , p. 21-27
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