Challenges in treating Pompe disease: an industry perspective
Author:
Publisher
AME Publishing Company
Subject
General Medicine
Cited by 43 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation;Molecular Genetics and Metabolism Reports;2024-12
2. Meeting Report: 2023 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Therapy of Neuromuscular Diseases’;Journal of Neuromuscular Diseases;2024-09-03
3. Neurological glycogen storage diseases and emerging therapeutics;Neurotherapeutics;2024-09
4. Clinical insight meets scientific innovation to develop a next generation ERT for Pompe disease;Molecular Genetics and Metabolism;2024-09
5. Cipaglucosidase alfa-atga: Unveiling new horizons in Pompe disease therapy;Health Sciences Review;2024-06
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