Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in subjects with mucopolysaccharidosis type VII < 5 years old

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

Long-term efficacy and safety of migalastat compared to enzyme replacement therapy in Fabry disease: Phase 3 study results

2015 ◽  
Vol 114 (2) ◽  
pp. S57 ◽  
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

Long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis type VII

2020 ◽  
Vol 129 (2) ◽  
pp. S158-S159
Author(s):  
Raymond Y. Wang ◽  
José Francisco da Silva Franco ◽  
Jaime López-Valdez ◽  
Esmeralda Martins ◽  
Vernon R. Sutton ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Mucopolysaccharidosis Type ◽  
Safety And Efficacy ◽  
Enzyme Replacement

Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy

2015 ◽  
Vol 114 (2) ◽  
pp. 129-137 ◽  
Author(s):  
Eveline J. Langereis ◽  
Naomi van Vlies ◽  
Heather J. Church ◽  
Ronald B. Geskus ◽  
Carla E.M. Hollak ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Enzyme Replacement
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