TY  - JOUR
AU  - Fernández-Simón, Esther
AU  - Carrasco-Rozas, Ana
AU  - Gallardo, Eduard
AU  - Figueroa-Bonaparte, Sebastián
AU  - Belmonte, Izaskun
AU  - Pedrosa, Irene
AU  - Montiel, Elena
AU  - Suárez-Calvet, Xavier
AU  - Alonso-Pérez, Jorge
AU  - Segovia, Sonia
AU  - Nuñez-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
UR  - http://hdl.handle.net/10668/13570
T2  - Scientific reports
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  - en
KW  - Adolescent
KW  - Adult
KW  - Becaplermin
KW  - Biomarkers
KW  - Case-Control Studies
KW  - Child
KW  - Female
KW  - Follow-Up Studies
KW  - Glycogen Storage Disease Type II
KW  - Humans
KW  - Male
KW  - Middle Aged
KW  - Muscle, Skeletal
KW  - Muscular Diseases
KW  - Prognosis
KW  - Prospective Studies
KW  - Young Adult
TI  - PDGF-BB serum levels are decreased in adult onset Pompe patients.
TY  - research article
VL  - 9
ER  -