A Race Against Time—Changing the Natural History of CRIM Negative Infantile Pompe Disease-Reference-Cited by-同舟云学术

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A Race Against Time—Changing the Natural History of CRIM Negative Infantile Pompe Disease

Published:2020-09-04 Issue: Volume:11 Page:
ISSN:1664-3224
Container-title:Frontiers in Immunology
language:
Short-container-title:Front. Immunol.

Author:

Gupta Punita,Shayota Brian J.,Desai Ankit K.,Kiblawi Fuad,Myridakis Dorothy,Messina John,Tah Peter,Tambini-King Lorien,Kishnani Priya S.

Publisher

Frontiers Media SA

Subject

Immunology,Immunology and Allergy

Reference31 articles.

1. Infantile-onset Pompe disease natural history study, a retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease;Kishnani;J Pediatr.,2006

2. The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature;van den Hout;Pediatrics,2003

3. Facial-muscle weakness, speech disorders and dysphagia are common in patients with classic infantile Pompe disease treated with enzyme therapy;van Gelder;J Inherit Metab Dis.,2012

4. CRIM-negative infantile Pompe disease: 42-month treatment outcome;Rohrbach;J Inherit Metab Dis.,2010

5. CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy;Berrier;Genet Med.,2015

Cited by 8 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. Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells;Molecular Therapy - Nucleic Acids;2024-06

3. Long-Term Outcome of Infantile Onset Pompe Disease Patients Treated with Enzyme Replacement Therapy - Data from a German-Austrian Cohort;Journal of Neuromuscular Diseases;2024-01-02

4. Infantile Pompe disease with intrauterine onset: a case report and literature review;Italian Journal of Pediatrics;2022-11-21

5. Hyperoxia combined with the B‐cell antagonist rituximab led to intestinal dysbiosis in neonatal mice;Microbiology and Immunology;2022-06-08

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