Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa-Reference-Cited by-同舟云学术

Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

Published:2012-11 Issue:3 Volume:107 Page:456-461
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

van der Ploeg Ans T.,Barohn Richard,Carlson Lisa,Charrow Joel,Clemens Paula R.,Hopkin Robert J.,Kishnani Priya S.,Laforêt Pascal,Morgan Claire,Nations Sharon,Pestronk Alan,Plotkin Horacio,Rosenbloom Barry E.,Sims Katherine B.,Tsao Elisa

Funder

Genzyme

Publisher

Elsevier BV

Subject

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

Reference12 articles.

1. Course of disability and respiratory function in untreated late onset Pompe disease;Hagemans;Neurology,2006

2. Glycogen Storage Disease Type II: acid alpha-glucosidase (acid maltase) deficiency;Hirschhorn,2001

3. Late-onset Pompe disease: clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients;Müller-Felber;Neuromuscul. Disord.,2007

4. Clinical features of late-onset Pompe disease: a prospective cohort study;Wokke;Muscle Nerve,2008

5. Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease;van der Beek;Neuromuscul. Disord.,2009

Cited by 93 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. High‐risk screening for late‐onset Pompe disease in China: An expanded multicenter study;Journal of Inherited Metabolic Disease;2024-09-03

2. Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials;Molecular Genetics and Metabolism Reports;2024-09

3. Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry;Journal of Neurology;2024-06-19

4. 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

5. 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