Fernández-Simón, Esther; Carrasco-Rozas, Ana; Gallardo, Eduard; Figueroa-Bonaparte, Sebastian; Belmonte, Izaskun; Pedrosa, Irene; Montiel, Elena; Suárez-Calvet, Xavier; Alonso-Pérez, Jorge; Segovia, Sonia; Nunez-Peralta, Claudia; Llauger, Jaume; Mayos, Mercedes; Illa, Isabel; Díaz-Manera, Jordi; Barba-Romero, Miguel-Ángel; Barcena, Joseba; Rosario-Carzorla, María; Creus, Carlota; Coll-Canti, Jaume; de-Luna, Noemi; Díaz, Manuel; Domínguez, Cristina; Fernández-Torron, Roberto; García-Antelo, María-José; Grau, Josep-María; Gómez-Caravaca, María-Teresa; Leon-Hernández, Juan-Carlos; López-de-Munain, Adolfo; Martínez-García, Francisco-Antonio; Morgado, Yolanda; Moreno, Antonio; Moris, German; Muñoz-Blanco, Miguel-Ángel; Nascimento, Andrés; Paradas, Carmen; Parajua-Pozo, José-Luis; Querol, Luis; Robledo-Strauss, Arturo; Rojas-García, Ricard; Rojas-Marcos, Inigo; Salazar, José-Antonio; Uson, Mercedes
(NATURE PORTFOLIO, 2019-02-14)
Adult onset Pompe disease is a genetic disorder characterized by slowly progressive skeletal and respiratory muscle weakness. Symptomatic patients are treated with enzymatic replacement therapy with human recombinant alfa ...