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Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2017.12.435
◽
2018
◽
Vol 123
(2)
◽
pp. 92-96
◽
Cited By ~ 3
Author(s):
Ankit K. Desai
◽
Crista K. Walters
◽
Heidi L. Cope
◽
Zoheb B. Kazi
◽
Stephanie M. DeArmey
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Clinical Experience
◽
Pompe Disease
◽
Enzyme Replacement
◽
Alglucosidase Alfa
Download Full-text
Related Documents
Cited By
References
A 4-year follow-up study of 24 patients with late onset Pompe disease treated with alglucosidase alfa enzyme replacement therapy at a single centre
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2014.12.252
◽
2015
◽
Vol 114
(2)
◽
pp. S111
Author(s):
Karolina M. Stepien
◽
Jane Whitby
◽
Mark Roberts
◽
Reena Sharma
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Single Centre
◽
Enzyme Replacement
◽
Follow Up Study
◽
Alglucosidase Alfa
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Long‐term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry
Journal of Inherited Metabolic Disease
◽
10.1002/jimd.12272
◽
2020
◽
Vol 43
(6)
◽
pp. 1219-1231
◽
Cited By ~ 1
Author(s):
Claudio Semplicini
◽
Marie De Antonio
◽
Nadjib Taouagh
◽
Anthony Béhin
◽
Françoise Bouhour
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Prospective Analysis
◽
Enzyme Replacement
◽
Alglucosidase Alfa
◽
Term Benefit
Download Full-text
Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2015.12.476
◽
2016
◽
Vol 117
(2)
◽
pp. S117-S118
Author(s):
Shafeeq Ladha
◽
Pascal Laforet
◽
Eugen Mengel
◽
Alan Pestronk
◽
Jean Pouget
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Phase 1
◽
The Novel
◽
Enzyme Replacement
◽
Alglucosidase Alfa
◽
Treatment Naïve
Download Full-text
Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients
Clinical Neurology and Neurosurgery
◽
10.1016/j.clineuro.2020.106048
◽
2020
◽
Vol 196
◽
pp. 106048
Author(s):
Chafic Karam
◽
Thomas Ragole
◽
Orly Moshe-Lilie
◽
Nizar Chahin
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Enzyme Replacement
◽
Alglucosidase Alfa
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Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2015.12.396
◽
2016
◽
Vol 117
(2)
◽
pp. S92
Author(s):
Loren Pena
◽
Richard Baron
◽
Barry Byrne
◽
Claude Desnuelle
◽
Ozlem Goker-Alpan
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Phase 1
◽
The Novel
◽
Enzyme Replacement
◽
Alglucosidase Alfa
◽
Treatment Naïve
Download Full-text
Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease
Orphanet Journal of Rare Diseases
◽
10.1186/1750-1172-9-75
◽
2014
◽
Vol 9
(1)
◽
pp. 75
◽
Cited By ~ 20
Author(s):
Tim A Kanters
◽
Iris Hoogenboom-Plug
◽
Maureen PMH Rutten-Van Mölken
◽
W Redekop
◽
Ans T van der Ploeg
◽
...
Keyword(s):
Cost Effectiveness
◽
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Enzyme Replacement
◽
Alglucosidase Alfa
Download Full-text
Strategy to assess the effect of duvoglustat co-administered with alglucosidase alfa infusion on the immune response to enzyme replacement therapy for Pompe disease
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2013.12.038
◽
2014
◽
Vol 111
(2)
◽
pp. S25
Author(s):
Elfrida R. Benjamin
◽
Xiaoyang Wu
◽
M. Cecilia Della Valle
◽
Eric Sjoberg
◽
Sheela Sitaraman
◽
...
Keyword(s):
Immune Response
◽
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Enzyme Replacement
◽
Alglucosidase Alfa
Download Full-text
Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2017.06.007
◽
2017
◽
Vol 122
(1-2)
◽
pp. 80-85
◽
Cited By ~ 14
Author(s):
Constantinos Papadopoulos
◽
David Orlikowski
◽
Hélène Prigent
◽
Arnaud Lacour
◽
Céline Tard
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Enzyme Replacement
◽
Alglucosidase Alfa
Download Full-text
Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease
Neuromuscular Disorders
◽
10.1016/j.nmd.2016.06.089
◽
2016
◽
Vol 26
◽
pp. S109-S110
◽
Cited By ~ 2
Author(s):
C. Papadopoulos
◽
D. Orlikowski
◽
H. Prigent
◽
B. Perniconi
◽
N. Taouagh
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Enzyme Replacement
◽
Alglucosidase Alfa
Download Full-text
36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy
Journal of Inherited Metabolic Disease
◽
10.1007/s10545-012-9451-8
◽
2012
◽
Vol 35
(5)
◽
pp. 837-845
◽
Cited By ~ 83
Author(s):
Caroline Regnery
◽
Cornelia Kornblum
◽
Frank Hanisch
◽
Stefan Vielhaber
◽
Nicola Strigl-Pill
◽
...
Keyword(s):
Clinical Study
◽
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Enzyme Replacement
◽
Alglucosidase Alfa
Download Full-text
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