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Enzyme Replacement Therapy for Pompe Disease: The Long-Term Efficacy and Limitation Tokiko Fukuda 1 , Hideo Sugie 2 1Pediatrics, Hamamatsu Medical University 2Faculty of Health and Medical Sciences, Tokoha University Keyword: ポンペ病 , 肢帯型筋ジストロフィー , 心肥大 , オートファジー , 交叉反応性免疫物質(CRIM) , Pompe disease , limb-girdle muscular dystrophy , cardiohypertrophy , autophagy , cross reactive immunological material pp.1091-1098
Published Date 2015/9/1
DOI https://doi.org/10.11477/mf.1416200265
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Abstract

The long-term efficacy of enzyme replacement therapy (ERT) in patients with Pompe disease has been unraveled. Cardiac muscle responds well to ERT, whereas motor and respiratory functions do not. The screening of newborns, which enables the early initiation of ERT, has improved outcomes of infantile Pompe patients. Myopathies still exist even in patients in whom ERT is initiated early, and have become recognized as an emerging new phenotype of IP on ERT. In addition, this shows the limitation of currently available ERT. It is expected that the development of therapy will improve the outcome of Pompe disease.


Copyright © 2015, Igaku-Shoin Ltd. All rights reserved.

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電子版ISSN 1344-8129 印刷版ISSN 1881-6096 医学書院

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