TY - GEN AU - Fernandez-Simon, Esther AU - Carrasco-Rozas, Ana AU - Gallardo, Eduard AU - Figueroa-Bonaparte, Sebastian AU - Belmonte, Izaskun AU - Pedrosa, Irene AU - Montiel, Elena AU - Suarez-Calvet, Xavier AU - Alonso-Perez, Jorge AU - Segovia, Sonia AU - Nunez-Peralta, Claudia AU - Llauger, Jaume AU - Mayos, Mercedes AU - Illa, Isabel AU - Spanish Pompe Study Group AU - Díaz-Manera, Jordi PY - 2019 DO - 10.1038/s41598-018-38025-0 SN - 2045-2322 UR - https://hdl.handle.net/20.500.12105/22774 AB - 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 glucosidase. Motor functional... LA - eng PB - Nature Publishing Group KW - Child KW - Case-Control Studies KW - Young Adult KW - Adult KW - Muscle, Skeletal KW - Follow-Up Studies KW - Glycogen Storage Disease Type II KW - Humans KW - Adolescent KW - Middle Aged KW - Prognosis KW - Muscular Diseases KW - Becaplermin KW - Male KW - Biomarkers KW - Prospective Studies KW - Female TI - PDGF-BB serum levels are decreased in adult onset Pompe patients TY - research article ER -