Blar i Bergen Open Research Archive på forfatter "Nowak, Albina"
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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
Hughes, Derralynn A.; Aguiar, Patricio; Deegan, Patrick B; Ezgu, Fatih; Frustaci, Andrea; Lidove, Olivier; Linhart, Aleš; Lubanda, Jean-Claude; Moon, James C; Nicholls, Kathleen; Niu, Dau-Ming; Nowak, Albina; Ramaswami, Uma; Reisin, Ricardo; Rozenfeld, Paula; Schiffmann, Raphael; Svarstad, Einar; Thomas, Mark; Torra, Roser; Vujkovac, Bojan; Warnock, David G.; West, Michael L; Johnson, Jack; Rolfe, Mark J; Feriozzi, Sandro (Journal article; Peer reviewed, 2020)Objectives 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 ... -
An expert consensus on the recommendations for the use of biomarkers in Fabry disease
Burlina, Allesandro; Brand, Eva; Hughes, Derralynn A.; Kantola, Ilkka; Krämer, Johannes; Nowak, Albina; Tøndel, Camilla; Wanner, Christoph; Spada, Marco (Journal article; Peer reviewed, 2023)Fabry disease is an X-linked lysosomal storage disorder caused by the accumulation of glycosphingolipids in various tissues and body fluids, leading to progressive organ damage and life-threatening complications. Phenotypic ... -
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; Goker-Alpan, Ozlem; Wilcox, William R; Holida, Myrl; Bernat, John; Longo, Nicola; Linhart, Aleš; Hughes, Derralynn A.; Hopkin, Robert; Tøndel, Camilla; Langeveld, Mirjam; Giraldo, Pilar; Pisani, Antonio; Germain, Dominique P.; Mehta, Ankit; Deegan, Patrick; Molnar, Maria Judith; Ortiz, Damara; Jovanovic, Ana; Muriello, Michael; Barshop, Bruce; 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 (Journal article; Peer reviewed, 2023)Background Pegunigalsidase alfa is a PEGylated α-galactosidase A enzyme replacement therapy. BALANCE (NCT02795676) assessed non-inferiority of pegunigalsidase alfa versus agalsidase beta in adults with Fabry disease with ... -
Systems analyses of the Fabry kidney transcriptome and its response to enzyme replacement therapy identified and cross-validated enzyme replacement therapy-resistant targets amenable to drug repurposing
Delaleu, Nicolas; Marti, Hans Peter; Strauss, Philipp; Sekulic, Miroslav; Osman, Tarig Al-Hadi; Tøndel, Camilla; Skrunes, Rannveig; Leh, Sabine; Svarstad, Einar; Nowak, Albina; Gaspert, Ariana; Rusu, Elena; Kwee, Ivo; Rinaldi, Andrea; Flatberg, Arnar; Eikrem, Øystein Solberg (Journal article; Peer reviewed, 2023)Fabry disease is a rare disorder caused by variations in the alpha-galactosidase gene. To a degree, Fabry disease is manageable via enzyme replacement therapy (ERT). By understanding the molecular basis of Fabry nephropathy ...