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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
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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
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◽
Single Centre
◽
Enzyme Replacement
◽
Follow Up Study
◽
Alglucosidase Alfa
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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
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
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
Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease
Journal of the Neurological Sciences
◽
10.1016/j.jns.2015.08.007
◽
2015
◽
Vol 358
(1-2)
◽
pp. 459-460
◽
Cited By ~ 1
Author(s):
J. Rafael Bretón Martínez
◽
Andrés Cánovas Martínez
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Enzyme Replacement
◽
Alglucosidase Alfa
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Enzyme replacement therapy with alglucosidase alfa in a late-onset Pompe disease patient during pregnancy
Neuromuscular Disorders
◽
10.1016/j.nmd.2018.08.002
◽
2018
◽
Vol 28
(11)
◽
pp. 965-968
◽
Cited By ~ 2
Author(s):
Miguel Oliveira Santos
◽
Teresinha Evangelista
◽
Isabel Conceição
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Disease Patient
◽
Enzyme Replacement
◽
Alglucosidase Alfa
Download Full-text
Reduced Efficacy of Enzyme Replacement Therapy in a Child with Late-Onset Pompe Disease
Pediatrics & Therapeutics
◽
10.4172/2161-0665.1000290
◽
2016
◽
Vol 06
(02)
◽
Cited By ~ 1
Author(s):
Satoru Takahashi
◽
Ryosuke Tanaka
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Enzyme Replacement
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FIVE-YEARS FOLLOW-UP OF THE RESPIRATORY AND MOTOR INVOLVEMENTS IN PATIENTS WITH LATE ONSET POMPE DISEASE (LOPD) ON IRREGULAR ENZYME REPLACEMENT THERAPY (ERT)
CHEST Journal
◽
10.1016/j.chest.2020.05.415
◽
2020
◽
Vol 157
(6)
◽
pp. A371
Author(s):
N. Sousa
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Enzyme Replacement
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Effects of enzyme replacement therapy on bone density in late onset Pompe disease
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.034
◽
2020
◽
Vol 129
(2)
◽
pp. S24-S25
Author(s):
Mahima Avanti
◽
Virginia Kimonis
◽
Alice Chen
Keyword(s):
Bone Density
◽
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Enzyme Replacement
Download Full-text
Obstructive sleep apnea in late-onset Pompe disease treated by enzyme replacement therapy
Neuromuscular Disorders
◽
10.1016/j.nmd.2020.02.004
◽
2020
◽
Vol 30
(4)
◽
pp. 329-330
Author(s):
Raj Bhui
◽
Andrew R. Spector
Keyword(s):
Obstructive Sleep Apnea
◽
Sleep Apnea
◽
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Pompe Disease
◽
Late Onset
◽
Enzyme Replacement
◽
Obstructive Sleep
Download Full-text
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