scholarly journals Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events

2018 ◽  
Vol 14 ◽  
pp. 31-35 ◽  
Author(s):  
Michael Beck ◽  
Derralynn Hughes ◽  
Christoph Kampmann ◽  
Guillem Pintos-Morell ◽  
Uma Ramaswami ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Long Term Outcomes ◽  
Enzyme Replacement ◽  
Agalsidase Alfa ◽  
Composite Events

scholarly journals Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events

2016 ◽  
Vol 117 (2) ◽  
pp. S25 ◽  
Author(s):  
Michael Beck ◽  
Derralynn Hughes ◽  
Christoph Kampmann ◽  
Svetlana Bizjajeva ◽  
Guillem Pintos-Morell ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Long Term Outcomes ◽  
Enzyme Replacement ◽  
Agalsidase Alfa ◽  
Composite Events

Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year Fabry Outcome Survey analysis

2016 ◽  
Vol 117 (2) ◽  
pp. S98 ◽  
Author(s):  
Uma Ramaswami ◽  
Michael Beck ◽  
Derralynn Hughes ◽  
Christoph Kampmann ◽  
Svetlana Bizjajeva ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Survey Analysis ◽  
Renal Outcomes ◽  
Enzyme Replacement ◽  
Agalsidase Alfa ◽  
Fabry Outcome Survey

scholarly journals Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors

PLoS ONE ◽  
2017 ◽  
Vol 12 (8) ◽  
pp. e0182379 ◽  
Author(s):  
Maarten Arends ◽  
Marieke Biegstraaten ◽  
Derralynn A. Hughes ◽  
Atul Mehta ◽  
Perry M. Elliott ◽  
...  
Keyword(s):  
Prognostic Factors ◽  
Retrospective Study ◽  
Enzyme Replacement Therapy ◽  
Fabry Disease ◽  
Replacement Therapy ◽  
Long Term Outcomes ◽  
Enzyme Replacement ◽  
Disease Analysis

scholarly journals Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy

2021 ◽  
Vol 133 (1) ◽  
pp. 100-108
Author(s):  
Paula Garcia ◽  
Dawn Phillips ◽  
JoAnn Johnson ◽  
Kenneth Martin ◽  
Linda M. Randolph ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Long Term Outcomes ◽  
Enzyme Replacement ◽  
Mucopolysaccharidosis Vi

scholarly journals Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis

2019 ◽  
Vol Volume 13 ◽  
pp. 3705-3715
Author(s):  
Uma Ramaswami ◽  
Michael Beck ◽  
Derralynn Hughes ◽  
Christoph Kampmann ◽  
Jaco Botha ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Renal Outcomes ◽  
Enzyme Replacement ◽  
Agalsidase Alfa ◽  
Fabry Outcome Survey

scholarly journals Correction: Barak, S. et al. “Long-Term Outcomes of Early Enzyme Replacement Therapy for Mucopolysaccharidosis IV: Clinical Case Studies of Two Siblings”. Diagnostics 2020, 10, 108

Diagnostics ◽  
2020 ◽  
Vol 10 (7) ◽  
pp. 480
Author(s):  
Sharon Barak ◽  
Yair Anikster ◽  
Ifat Sarouk ◽  
Eve Stern ◽  
Etzyona Eisenstein ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Case Studies ◽  
Clinical Case ◽  
Long Term Outcomes ◽  
Enzyme Replacement ◽  
Clinical Case Studies

The authors wish to make the following correction to this paper [...]

Two siblings with attenuated MPS II form: Long term enzyme replacement therapy

2021 ◽  
Vol 132 (2) ◽  
pp. S107-S108
Author(s):  
Nato Vashakmadze ◽  
Leyla Namazova-Baranova ◽  
Natalia Zhurkova ◽  
Olga Gordeeva ◽  
Nina Fedorova ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Enzyme Replacement ◽  
Mps Ii
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