The new era of Pompe disease: Advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management-Reference-Cited by-同舟云学术

The new era of Pompe disease: Advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management

Published:2012-01-17 Issue:1 Volume:160C Page:1-7
ISSN:1552-4868
Container-title:American Journal of Medical Genetics Part C: Seminars in Medical Genetics
language:en
Short-container-title:Am. J. Med. Genet.

Author:

Kishnani Priya S.,Beckemeyer Alexandra A.,Mendelsohn Nancy J.

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Reference34 articles.

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4. Whole-body muscle MRI in 20 patients suffering from late onset Pompe disease: Involvement patterns;Carlier;Neuromuscul Disord,2011

5. Brain development in infantile-onset Pompe disease treated by enzyme replacement therapy;Chien;Pediatr Res,2006

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1. Real-world evidence study finds no new-onset diabetes or drug-related hyperglycemia in Pompe disease patients treated with avalglucosidase alfa;Molecular Genetics and Metabolism Reports;2024-03

2. Безопасность и эффективность авалглюкозидазы альфа по сравнению с алглюкозидазой альфа у пациентов с болезнью Помпе с поздним началом: рандомизированное многоцентровое исследование III фазы COMET;Neuromuscular Diseases;2024-01-05

3. Exploring the Therapeutic Potential of Avalglucosidase Alfa in Infantile-Onset Pompe Disease with High Anti-Alglucosidase Alfa Antibody Titers;2024

4. Therapeutic Options for the Management of Pompe Disease: Current Challenges and Clinical Evidence in Therapeutics and Clinical Risk Management;Therapeutics and Clinical Risk Management;2022-12

5. Newborn screening for Pompe disease: Parental experiences and follow‐up care for a late‐onset diagnosis;Journal of Genetic Counseling;2022-08