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Orphanet Journal of Rare Diseases
|
August 26, 2017
A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
Barry J Byrne, Tarekegn Geberhiwot, Bruce A Barshop, et al.
Journal of Neurology
|
June 19, 2024
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
Kenneth I Berger, Yin-Hsiu Chien, Alberto Dubrovsky, et al.
Oncotarget
|
October 4, 2017
BPTF inhibits NK cell activity and the abundance of natural cytotoxicity receptor co-ligands
Kimberly Mayes, Zeinab Elsayed, Aiman Alhazmi, et al.
Journal of Neurology
|
July 31, 2025
Correction: 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
Kenneth I Berger, Yin-Hsiu Chien, Alberto Dubrovsky, et al.
Journal of Patient-Reported Outcomes
|
November 13, 2024
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Priya S Kishnani, Barry J Byrne, Kristl G Claeys, et al.
Current Biology : CB
|
April 30, 2025
Genetic basis of an adaptive polymorphism controlling butterfly silver iridescence
Luca Livraghi, Joseph J Hanly, Ling S Loh, et al.
Neuromuscular Disorders : NMD
|
February 8, 2011
Mosaic caveolin-3 expression in acquired rippling muscle disease without evidence of myasthenia gravis or acetylcholine receptor autoantibodies
Harriet P Lo, Enrico Bertini, Massimiliano Mirabella, et al.
Progress in Neurobiology
|
February 1, 2020
White matter structure and myelin-related gene expression alterations with experience in adult rats
Cassandra Sampaio-Baptista, Astrid Vallès, Alexandre A Khrapitchev, et al.
The Lancet. Neurology
|
November 20, 2021
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial
Benedikt Schoser, Mark Roberts, Barry J Byrne, et al.
Muscle & Nerve
|
October 21, 2017
Chronic pain has a strong impact on quality of life in facioscapulohumeral muscular dystrophy
Germán Morís, Libby Wood, Roberto FernáNdez-Torrón, et al.
Page
of 19
Search research articles
Search
Showing results (141-150 of 189) with videos related to
Sort By:
Page
of 19
Orphanet Journal of Rare Diseases
|
August 26, 2017
A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
Barry J Byrne, Tarekegn Geberhiwot, Bruce A Barshop, et al.
Journal of Neurology
|
June 19, 2024
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
Kenneth I Berger, Yin-Hsiu Chien, Alberto Dubrovsky, et al.
Oncotarget
|
October 4, 2017
BPTF inhibits NK cell activity and the abundance of natural cytotoxicity receptor co-ligands
Kimberly Mayes, Zeinab Elsayed, Aiman Alhazmi, et al.
Journal of Neurology
|
July 31, 2025
Correction: 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
Kenneth I Berger, Yin-Hsiu Chien, Alberto Dubrovsky, et al.
Journal of Patient-Reported Outcomes
|
November 13, 2024
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Priya S Kishnani, Barry J Byrne, Kristl G Claeys, et al.
Current Biology : CB
|
April 30, 2025
Genetic basis of an adaptive polymorphism controlling butterfly silver iridescence
Luca Livraghi, Joseph J Hanly, Ling S Loh, et al.
Neuromuscular Disorders : NMD
|
February 8, 2011
Mosaic caveolin-3 expression in acquired rippling muscle disease without evidence of myasthenia gravis or acetylcholine receptor autoantibodies
Harriet P Lo, Enrico Bertini, Massimiliano Mirabella, et al.
Progress in Neurobiology
|
February 1, 2020
White matter structure and myelin-related gene expression alterations with experience in adult rats
Cassandra Sampaio-Baptista, Astrid Vallès, Alexandre A Khrapitchev, et al.
The Lancet. Neurology
|
November 20, 2021
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial
Benedikt Schoser, Mark Roberts, Barry J Byrne, et al.
Muscle & Nerve
|
October 21, 2017
Chronic pain has a strong impact on quality of life in facioscapulohumeral muscular dystrophy
Germán Morís, Libby Wood, Roberto FernáNdez-Torrón, et al.
Page
of 19