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- Title
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II.
- Authors
Bembi, Bruno; Pisa, Federica Edith; Confalonieri, Marco; Ciana, Giovanni; Fiumara, Agata; Parini, Rossella; Rigoldi, Miriam; Moglia, Arrigo; Costa, Alfredo; Carlucci, Annalisa; Danesino, Cesare; Pittis, Maria Gabriela; Dardis, Andrea; Ravaglia, Sabrina
- Abstract
Type II glycogenosis (GSDII) is a lysosomal storage disorder due to acid alpha-glucosidase (GAA) deficiency. Enzyme replacement therapy (ERT) with human recombinant alpha-glucosidase (rhGAA) has been demonstrated to be effective in the treatment of infantile forms of GSDII, but little information is available concerning late-onset phenotypes. Long-term follow-up studies are not available at present. The aim of this study was to evaluate the ERT long-term effects in late-onset GSDII.
- Publication
Journal of inherited metabolic disease, 2010, Vol 33, Issue 6, p727
- ISSN
1573-2665
- Publication type
Journal Article
- DOI
10.1007/s10545-010-9201-8