Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease-Reference-Cited by-同舟云学术

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Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease

Published:2020-02 Issue:2 Volume:129 Page:67-72
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Koeberl Dwight D.,Case Laura E.,Desai Ankit,Smith Edward C.,Walters Crista,Han Sang-oh,Thurberg Beth L.,Young Sarah P.,Bali Deeksha,Kishnani Priya S.

Funder

Genzyme Sanofi

Sanofi Genzyme

Alice and Y.-T. Chen Pediatric Genetics and Genomics Center

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference23 articles.

1. The emerging phenotype of long-term survivors with infantile Pompe disease;Prater;Genet Med.,2012

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

3. Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme(R)) in 12 patients with advanced late-onset Pompe disease;Papadopoulos;Mol. Genet. Metab.,2017

4. Long-term benefit of enzyme replacement therapy in Pompe disease: a 5-year prospective study;Kuperus;Neurology.,2017

5. Enzyme replacement therapy in the mouse model of Pompe disease;Raben;Mol. Genet. Metab.,2003

Cited by 13 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. Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease;Orphanet Journal of Rare Diseases;2024-01-12

2. Enzyme replacement therapy for late-onset Pompe disease;Cochrane Database of Systematic Reviews;2023-12-12

3. Salbutamol ameliorates skeletal muscle wasting and inflammatory markers in streptozotocin (STZ)-induced diabetic rats;International Immunopharmacology;2023-11

4. Glycogen storage diseases;Nature Reviews Disease Primers;2023-09-07

5. Hypertrophic Cardiomyopathy versus Storage Diseases with Myocardial Involvement;International Journal of Molecular Sciences;2023-08-26

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