scholarly journals Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age

2020 ◽  
Vol Volume 14 ◽  
pp. 2149-2158
Author(s):  
Rossella Parini ◽  
Guillem Pintos-Morell ◽  
Julia B Hennermann ◽  
Ting-Rong Hsu ◽  
Nesrin Karabul ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Enzyme Replacement ◽  
Cardiac Outcomes ◽  
Agalsidase Alfa ◽  
Before And After ◽  
Fabry Outcome Survey

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 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 ADULT-ONSET HYPOPHOSPHATASIA: BEFORE AND AFTER TREATMENT WITH ASFOTASE ALFA

2019 ◽  
Vol 5 (6) ◽  
pp. e344-e348
Author(s):  
Angela L. Magdaleno ◽  
Sonum Singh ◽  
Sandhya Venkataraman ◽  
Gretchen A. Perilli ◽  
Ya-Yu Lee
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Laboratory Data ◽  
Whole Body ◽  
Adult Onset ◽  
Enzyme Replacement ◽  
Asfotase Alfa ◽  
Focal Uptake ◽  
Patient Reported ◽  
Before And After

Objective: To review the diagnosis and clinical course of a woman with hypophosphatasia who is being treated with newly approved enzyme replacement therapy, asfotase alfa. Methods: Clinical and laboratory data are presented. Results: This is a unique report of a woman with debilitating adult-onset hypophosphatasia who was successfully diagnosed with low alkaline phosphatase (ALP) levels and elevated vitamin B6 levels. Treatment with asfotase alfa resolved her chronic bony pain symptoms and quadrupled her daily pedometer step count. Furthermore, whole body scans before and after treatment showed less focal uptake overall, suggesting fracture healing after enzyme replacement therapy. Conclusion: Improvement in patient reported symptoms, daily pedometer count, and whole body scans was noted after treatment of adult-onset hypophosphatasia with asfotase alfa enzyme replacement therapy. The significance of increased ALP levels after treatment is currently unknown.

Enzyme replacement therapy in Fabry disease: Comparison of agalsidase alfa and agalsidase beta

2008 ◽  
Vol 95 (1-2) ◽  
pp. 114-115 ◽  
Author(s):  
Atul Mehta ◽  
Michael Beck ◽  
Christoph Kampmann ◽  
Andrea Frustaci ◽  
Dominique P. Germain ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Fabry Disease ◽  
Replacement Therapy ◽  
Agalsidase Beta ◽  
Enzyme Replacement ◽  
Agalsidase Alfa
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