Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy

Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy

Abstract 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...

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Bibliographic Details
Main Authors: Chien-Yu Hsueh, Chii-Yuan Huang, Chia-Feng Yang, Chia-Chen Chang, Wei-Sheng Lin, Hsiu-Lien Cheng, Shang-Liang Wu, Yen-Fu Cheng, Dau-Ming Niu
Format: Article
Language:English
Published: BMC 2021-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Infantile-onset Pompe disease
Enzyme-replacement therapy
Hearing loss
Congenital deafness
Lysosomal storage disorder
Glycogen storage disorder
Online Access:https://doi.org/10.1186/s13023-021-01817-1

Internet

https://doi.org/10.1186/s13023-021-01817-1

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