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Neuroimaging Findings in Infantile Pompe Patients Treated with Enzyme Replacement Therapy
BACKGROUND: Recombinant human acid α-glucosidase (rhGAA) enzyme replacement therapy (ERT) has prolonged survival in infantile Pompe disease (IPD), but has unmasked central nervous system (CNS) changes. METHODS: Brain imaging, consisting of computed tomography (CT) and/or magnetic resonance imaging (...
Gorde:
| Argitaratua izan da: | Mol Genet Metab |
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| Egile Nagusiak: | , , , , , , , , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
2017
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5808895/ https://ncbi.nlm.nih.gov/pubmed/29050825 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.10.005 |
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