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 ...