Blar i Bergen Open Research Archive på forfatter "Feldt-Rasmussen, Ulla"
-
Clinical outcomes among young patients with Fabry disease who initiated agalsidase beta treatment before 30 years of age: An analysis from the Fabry registry
Hopkin, Robert J.; Cabrera, Gustavo; Jefferies, John; Yang, Meng; Ponce, Elvira; Brand, Eva; Feldt-Rasmussen, Ulla; Germain, Dominique P.; Guffon, Nathalie; Jovanovic, Ana; Kantola, Ilkka; Karaa, Amel; Martins, Ana; Tøndel, Camilla; Wilcox, William R; Yoo, Han-Wook; Burlina, Alessandro; Mauer, Michael (Journal article; Peer reviewed, 2023)Background Clinical manifestations of classic Fabry disease (α-galactosidase A deficiency) usually occur in childhood, while complications involving major organs typically develop in adulthood. Outcomes of Fabry-specific ... -
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document
Biegstraaten, Marieke; Arngrímsson, R; Barbey, F; Boks, L; Cecchi, Franco; Deegan, PB; Feldt-Rasmussen, Ulla; Geberhiwot, T; Germain, DP; Hendriksz, C; Hughes, DA; Kantola, I; Karabul, N; Lavery, C; Linthorst, GE; Mehta, A; van de Mheen, E; Oliveira, JP; Parini, R; Ramaswami, Uma; Rudnicki, M; Serra, A; Sommer, Claudia; Sunder-Plassmann, G; Svarstad, Einar; Sweeb, A; Terryn, W; Tylki-Szymanska, A; Tøndel, Camilla; Vujkovac, B; Weidemann, Frank; Wijburg, FA; Woolfson, P; Hollak, CE. (Peer reviewed; Journal article, 2015-05-27)Introduction Fabry disease (FD) is a lysosomal storage disorder resulting in progressive nervous system, kidney and heart disease. Enzyme replacement therapy (ERT) may halt or attenuate disease progression. Since administration ...