Results 11 to 20 of about 167,688 (127)

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

Drug Design, Development and Therapy, 2019
Uma Ramaswami,1 Michael Beck,2 Derralynn Hughes,1 Christoph Kampmann,2 Jaco Botha,3 Guillem Pintos-Morell,4 Michael L West,5 Dau-Ming Niu,6 Kathy Nicholls,7,8 Roberto Giugliani9 On behalf of the FOS Study Group 1Royal Free London NHS Foundation
Ramaswami U, Beck M, Hughes D, Kampmann C, Botha J, Pintos-Morell G, West ML, Niu DM, Nicholls K, Giugliani R   +9 more
doaj   +4 more sources

Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis [PDF]

Molecular Genetics and Metabolism Reports, 2015
Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease.
Michael Beck, Derralynn Hughes, Christoph Kampmann, Sylvain Larroque, Atul Mehta, Guillem Pintos-Morell, Uma Ramaswami, Michael West, Anna Wijatyk, Roberto Giugliani   +9 more
doaj   +4 more sources

Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy was underpowered [PDF]

Drug Design, Development and Therapy, 2015
Alberto Ortiz,1,2 Maria Dolores Sanchez-Niño1,2 1Department of Nephrology, IIS-Fundacion Jimenez Diaz, School of Medicine, Universidad Autónoma de Madrid, 2Department of Nephrology, Instituto Reina Sofia de Investigación Nefrol ...
Ortiz A, Sanchez-Niño MD
doaj   +3 more sources

Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. [PDF]

PLoS ONE, 2007
Two different enzyme preparations, agalsidase alfa (Replagal(TM), Shire) and beta (Fabrazyme(TM), Genzyme), are registered for treatment of Fabry disease.
Anouk C Vedder, Gabor E Linthorst, Gunnar Houge, Johannna E M Groener, Els E Ormel, Berto J Bouma, Johannes M F G Aerts, Asle Hirth, Carla E M Hollak   +8 more
doaj   +2 more sources

Long-term effectiveness and safety outcomes in adults with Fabry disease treated with agalsidase alfa: 20 years of data from the Fabry Outcome Survey. [PDF]

Eur J Clin Invest
Background: We present the final report from the Fabry Outcome Survey (FOS) on long-term effectiveness and safety of agalsidase alfa in adults (≥18 years old). Methods: FOS was an international, multicentre, observational registry (NCT03289065), designed
Hughes DA, Pintos-Morell G, Kampmann C, Anagnostopoulou C, Botha J, Jain S, Nicholls K, Niu DM, Reisin R, West ML, Schenk J, Ramaswami U, Giugliani R.   +12 more
europepmc   +3 more sources

A rapid method to reduce drug interferences for antibody measurements in pegunigalsidase alfa-treated patients with Fabry disease [PDF]

Frontiers in Immunology
AimNeutralizing anti-drug antibodies (ADA) limit therapy efficacies significantly. Pegunigalsidase alfa is a newly approved drug for the treatment of Fabry disease.
Malte Lenders, Elisa Rudolph, Michael Rudnicki, Markus Cybulla, Eva Brand   +4 more
doaj   +2 more sources

Update on role of agalsidase alfa in management of Fabry disease [PDF]

Drug Design, Development and Therapy, 2011
Uma RamaswamiPaediatric Metabolic Unit, Cambridge University Hospitals, Cambridge, UKAbstract: Fabry disease (FD) is an X-linked lysosomal storage disorder that affects both men and women.
Uma Ramaswami
doaj   +2 more sources

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 [PDF]

Drug Design, Development and Therapy, 2020
Rossella Parini,1,2 Guillem Pintos-Morell,3 Julia B Hennermann,4 Ting-Rong Hsu,5 Nesrin Karabul,6 Vasiliki Kalampoki,7 Andrey Gurevich,7 Uma Ramaswami8 On behalf of the FOS Study Group1Rare Metabolic Diseases Unit, MBBM Foundation, San Gerardo Hospital ...
Parini R, Pintos-Morell G, Hennermann JB, Hsu TR, Karabul N, Kalampoki V, Gurevich A, Ramaswami U   +7 more
doaj   +2 more sources

Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study. [PDF]

J Med Genet, 2018
Two recombinant enzymes (agalsidase alfa 0.2 mg/kg/every other week and agalsidase beta 1.0 mg/kg/every other week) have been registered for the treatment of Fabry disease (FD), at equal high costs.
Arends M, Biegstraaten M, Wanner C, Sirrs S, Mehta A, Elliott PM, Oder D, Watkinson OT, Bichet DG, Khan A, Iwanochko M, Vaz FM, van Kuilenburg ABP, West ML, Hughes DA, Hollak CEM.   +15 more
europepmc   +5 more sources

Evaluating the relationship between antidrug antibodies and efficacy and safety outcomes in patients with Fabry disease receiving enzyme replacement therapy: a systematic literature review [PDF]

Orphanet Journal of Rare Diseases
Background Fabry disease is a lysosomal storage disorder caused by deficient activity of the enzyme α-galactosidase A, resulting in progressive accumulation of globotriaosylceramide (Gb3) and widespread tissue and organ damage. Enzyme replacement therapy
Patricio Aguiar, John A. Bernat, Derralynn Hughes   +2 more
doaj   +2 more sources