Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

Abstract Background Enzyme replacement therapy (ERT) has deeply modified the clinical history of Infantile Onset Pompe Disease (IOPD). However, its long-term effectiveness is still not completely defined. Available data shows a close relationship between clinical outcome and patients’ cross-reactive...

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Bibliographic Details
Main Authors: Rossella Parini, Paola De Lorenzo, Andrea Dardis, Alberto Burlina, Alessandra Cassio, Paolo Cavarzere, Daniela Concolino, Roberto Della Casa, Federica Deodato, Maria Alice Donati, Agata Fiumara, Serena Gasperini, Francesca Menni, Veronica Pagliardini, Michele Sacchini, Marco Spada, Roberta Taurisano, Maria Grazia Valsecchi, Maja Di Rocco, Bruno Bembi
Format: Article
Language:English
Published: BMC 2018-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Infantile onset Pompe disease
Alglucosidase alpha
ERT
Recombinant human GAA
rhGAA
Online Access:http://link.springer.com/article/10.1186/s13023-018-0771-0

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http://link.springer.com/article/10.1186/s13023-018-0771-0

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