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Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy
BACKGROUND: Studies suggest that enzyme-replacement therapy (ERT) is crucial to the survival of patients with infantile-onset Pompe disease (IOPD). Hearing impairment (HI) is one of the clinical sequelae observed in long-term survivors. However, the benefits of early ERT for hearing outcomes have no...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8340467/ https://www.ncbi.nlm.nih.gov/pubmed/34353347 http://dx.doi.org/10.1186/s13023-021-01817-1 |