ScienceGate
Advanced Search
Author Search
Journal Finder
Blog
Sign in / Sign up
ScienceGate
Search
Author Search
Journal Finder
Blog
Sign in / Sign up
Long-term efficacy and safety of migalastat compared to enzyme replacement therapy in Fabry disease: Phase 3 study results
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2014.12.118
◽
2015
◽
Vol 114
(2)
◽
pp. S57
◽
Cited By ~ 3
Author(s):
Derralynn Hughes
◽
Daniel G. Bichet
◽
Roberto Giugliani
◽
Raphael Schiffmann
◽
William R. Wilcox
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Fabry Disease
◽
Replacement Therapy
◽
Efficacy And Safety
◽
Phase 3
◽
Enzyme Replacement
◽
Term Efficacy
◽
Study Results
Download Full-text
Related Documents
Cited By
References
Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in subjects with mucopolysaccharidosis type VII < 5 years old
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.239
◽
2020
◽
Vol 129
(2)
◽
pp. S95
Author(s):
Heather Lau
◽
David Viskochil
◽
Pranoot Tanpaiboon
◽
Antonio Gonzalez-Meneses Lopez
◽
Esmeralda Martins
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Mucopolysaccharidosis Type
◽
Efficacy And Safety
◽
Enzyme Replacement
◽
Term Efficacy
Download Full-text
Results of a global phase 3, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of sebelipase alfa as an enzyme replacement therapy in children and adults with lysosomal acid lipase deficiency
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2014.12.042
◽
2015
◽
Vol 114
(2)
◽
pp. S27
Author(s):
Barbara Burton
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Controlled Trial
◽
Efficacy And Safety
◽
Acid Lipase
◽
Phase 3
◽
Double Blind
◽
Double Blind Placebo
◽
Lysosomal Acid
◽
Enzyme Replacement
Download Full-text
Long-Term Effects of Enzyme Replacement Therapy for Anderson-Fabry Disease
International Heart Journal
◽
10.1536/ihj.17-688
◽
2019
◽
Vol 60
(1)
◽
pp. 208-214
Author(s):
Miki Tsujiuchi
◽
Mio Ebato
◽
Hideyuki Maezawa
◽
Takuya Mizukami
◽
Ayaka Nogi
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Fabry Disease
◽
Replacement Therapy
◽
Long Term Effects
◽
Enzyme Replacement
Download Full-text
Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study
American Journal of Medical Genetics Part A
◽
10.1002/ajmg.a.61105
◽
2019
◽
Vol 179
(6)
◽
pp. 1069-1073
◽
Cited By ~ 3
Author(s):
Derralynn A. Hughes
◽
Kathleen Nicholls
◽
Gere Sunder‐Plassmann
◽
Ana Jovanovic
◽
Ulla Feldt‐Rasmussen
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Fabry Disease
◽
Replacement Therapy
◽
Phase 3
◽
Enzyme Replacement
◽
Disease Experience
Download Full-text
Long-term enzyme replacement therapy is associated with reduced proteinuria and preserved proximal tubular function in women with Fabry disease
Nephrology Dialysis Transplantation
◽
10.1093/ndt/gft452
◽
2013
◽
Vol 29
(3)
◽
pp. 619-625
◽
Cited By ~ 15
Author(s):
T. Prabakaran
◽
H. Birn
◽
B. M. Bibby
◽
A. Regeniter
◽
S. S. Sorensen
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Fabry Disease
◽
Replacement Therapy
◽
Tubular Function
◽
Enzyme Replacement
◽
Proximal Tubular
◽
Proximal Tubular Function
Download Full-text
Long term enzyme replacement therapy for Fabry disease: effectiveness on heart, kidney and brain
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2016.11.058
◽
2017
◽
Vol 120
(1-2)
◽
pp. S33
Author(s):
Gustavo H Cabrera
◽
Juan Manuel Politei
Keyword(s):
Enzyme Replacement Therapy
◽
Fabry Disease
◽
Replacement Therapy
◽
Enzyme Replacement
Download Full-text
Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study
Journal of Inherited Metabolic Disease
◽
10.1007/s10545-014-9715-6
◽
2014
◽
Vol 37
(6)
◽
pp. 979-990
◽
Cited By ~ 125
Author(s):
Christian J. Hendriksz
◽
◽
Barbara Burton
◽
Thomas R. Fleming
◽
Paul Harmatz
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Controlled Study
◽
Efficacy And Safety
◽
Phase 3
◽
Mucopolysaccharidosis Iva
◽
Enzyme Replacement
◽
Morquio A
◽
Morquio A Syndrome
◽
Elosulfase Alfa
Download Full-text
Long-term efficacy and safety of secukinumab in patients with psoriatic arthritis: 5-year (end-of-study) results from the phase 3 FUTURE 2 study
The Lancet Rheumatology
◽
10.1016/s2665-9913(20)30036-9
◽
2020
◽
Vol 2
(4)
◽
pp. e227-e235
◽
Cited By ~ 2
Author(s):
Iain B McInnes
◽
Philip J Mease
◽
Alan J Kivitz
◽
Peter Nash
◽
Proton Rahman
◽
...
Keyword(s):
Psoriatic Arthritis
◽
Efficacy And Safety
◽
Phase 3
◽
Term Efficacy
◽
Study Results
◽
Future 2
Download Full-text
Long-term efficacy of enzyme replacement therapy for Adenosine deaminase (ADA)-deficient Severe Combined Immunodeficiency (SCID)
Clinical Immunology
◽
10.1016/j.clim.2005.07.006
◽
2005
◽
Vol 117
(2)
◽
pp. 133-143
◽
Cited By ~ 107
Author(s):
Belinda Chan
◽
Diane Wara
◽
John Bastian
◽
Michael S. Hershfield
◽
John Bohnsack
◽
...
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Adenosine Deaminase
◽
Severe Combined Immunodeficiency
◽
Combined Immunodeficiency
◽
Enzyme Replacement
◽
Term Efficacy
Download Full-text
Long-term Effect of Enzyme Replacement Therapy (ERT) for Fabry Disease on Diastolic Function as Manifested by Left Atrial (LA) Volumes
Heart Lung and Circulation
◽
10.1016/j.hlc.2010.06.456
◽
2010
◽
Vol 19
◽
pp. S188
Author(s):
A. Boyd
◽
N. Sadick
◽
D. Silence
◽
L. Thomas
Keyword(s):
Enzyme Replacement Therapy
◽
Fabry Disease
◽
Replacement Therapy
◽
Diastolic Function
◽
Left Atrial
◽
Term Effect
◽
Enzyme Replacement
◽
Long Term Effect
Download Full-text
Sign in / Sign up
Close
Export Citation Format
Close
Share Document
Close