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

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 immunol...

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發表在:Orphanet J Rare Dis
Main Authors: Parini, Rossella, De Lorenzo, Paola, Dardis, Andrea, Burlina, Alberto, Cassio, Alessandra, Cavarzere, Paolo, Concolino, Daniela, Della Casa, Roberto, Deodato, Federica, Donati, Maria Alice, Fiumara, Agata, Gasperini, Serena, Menni, Francesca, Pagliardini, Veronica, Sacchini, Michele, Spada, Marco, Taurisano, Roberta, Valsecchi, Maria Grazia, Di Rocco, Maja, Bembi, Bruno
格式: Artigo
語言:Inglês
出版: BioMed Central 2018
主題:
Research
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5806382/
https://ncbi.nlm.nih.gov/pubmed/29422078
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0771-0
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