Variable clinical features and genotype-phenotype correlations in 18 patients with late-onset Pompe disease-Reference-Cited by-同舟云学术

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Variable clinical features and genotype-phenotype correlations in 18 patients with late-onset Pompe disease

Published:2019-07 Issue:13 Volume:7 Page:276-276
ISSN:2305-5839
Container-title:Annals of Translational Medicine
language:
Short-container-title:Ann. Transl. Med

Author:

Alandy-dy Jousef,Wencel Marie,Hall Kathy,Simon Julie,Chen Yanjun,Valenti Erik,Yang Jade,Bali Deeksha,Lakatos Anita,Goyal Namita,Mozaffar Tahseen,Kimonis Virginia

Publisher

AME Publishing Company

Subject

General Medicine

Cited by 16 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. Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease;2024-02-22

2. Effects of enzyme replacement therapy on bone density in late onset Pompe disease;Molecular Genetics and Metabolism;2023-11

3. Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review;Biomolecules;2023-09-19

4. Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes;Biology;2023-08-23

5. Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry;Molecular Genetics and Metabolism;2023-07

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