Long-term observation of patients with advanced late-onset Pompe disease undergoing enzyme replacement therapy: A 15-year observation in a single center-Reference-Cited by-同舟云学术

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Long-term observation of patients with advanced late-onset Pompe disease undergoing enzyme replacement therapy: A 15-year observation in a single center

Published:2024-08 Issue: Volume: Page:
ISSN:0387-7604
Container-title:Brain and Development
language:en
Short-container-title:Brain and Development

Author:

Mori-Yoshimura Madoka,Takizawa Hotake,Unuma Atsushi,Oya Yasushi,Yorimoto Keisuke,Katsuta Wakana,Miyagi Kenji,Sato Noriko,Hara Takatoshi,Takahashi Yuji

Funder

National Center of Neurology and Psychiatry

Publisher

Elsevier BV

Reference19 articles.

1. Glycogen storage disease type II; acid alpha-glycosidase (acid maltase) deficiency;Hirshhorn,2001

2. Acid maltase deficiency;Engel,2004

3. A systematic review and Meta-analysis of enzyme replacement therapy in late-onset Pompe disease;Dornelles;J Clin Med,2021

4. Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review;Toscano;J Neurol,2013

5. Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis;Schoser;J Neurol,2017

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