Results 11 to 20 of about 744,838 (140)

Clinical and Biochemical Improvement After Switching From Agalsidase Alfa to Beta in a Boy With Classic Fabry Disease: A Case Report [PDF]

Clinical Case Reports
We described a 12‐year‐old boy with classic Fabry disease who was diagnosed through newborn screening. At age 6.2, he started agalsidase alfa based on evidence of subclinical organ involvement. At age 7.2, acroparesthesia subsequently developed.
Nobuhiko Koga, Hiroaki Yodogawa, Takaaki Sawada, Yuichi Mushimoto, Shinichiro Nagamitsu, Kimitoshi Nakamura, Shinichi Hirose, Takahito Inoue   +7 more
doaj   +4 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

Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients [PDF]

Orphanet Journal of Rare Diseases, 2011
Background Enzyme replacement therapy is currently the only approved therapy for Fabry disease. From June 2009 on, viral contamination of Genzyme's production facility resulted in a worldwide shortage of agalsidase beta leading to involuntary dose ...
Hollak Carla EM, Poorthuis Ben JHM, Overkleeft Hermen S, Mirzaian Mina, Kuiper Symen, Aerts Johannes MFG, Rombach Saskia M, Smid Bouwien E, Groener Johanna EM, Linthorst Gabor E   +9 more
doaj   +3 more sources

Safety and tolerability of agalsidase beta infusions shorter than 90 min in patients with Fabry disease: post-hoc analysis of a Japanese post-marketing study [PDF]

Orphanet Journal of Rare Diseases, 2023
Background Agalsidase beta, an enzyme replacement therapy for Fabry disease, is dosed biweekly at 1 mg/kg body weight, with increasing infusion rates based on tolerability.
Chae Sung Lee, Mina Tsurumi, Yoshikatsu Eto   +2 more
doaj   +2 more sources

A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and agalsidase beta [PDF]

Orphanet Journal of Rare Diseases
Background Fabry disease (FD) is a rare inherited X-linked lysosomal disorder caused by the deficiency or dysfunction of the enzyme α-galactosidase.
Ian Keyzor, Ana Maria Martins, Sema Kalkan Uçar, Hiroyuki Yamakawa, Yin-Hsiu Chien, Nur Arslan, Dau-Ming Niu, Leyla Tümer, Laura Baldock, Simon Shohet, Joseph D. Giuliano   +10 more
doaj   +2 more sources

Improvement of gastrointestinal symptoms in a significant proportion of male patients with classic Fabry disease treated with agalsidase beta: A Fabry Registry analysis stratified by phenotype [PDF]

Molecular Genetics and Metabolism Reports, 2020
Background: Fabry disease is an inherited disorder of glycolipid metabolism with progressive involvement of multiple organs, including the gastrointestinal tract, in classically affected male patients.
Robert J. Hopkin, Ulla Feldt-Rasmussen, Dominique P. Germain, Ana Jovanovic, Ana Maria Martins, Kathleen Nicholls, Alberto Ortiz, Juan Politei, Elvira Ponce, Carmen Varas, Frank Weidemann, Meng Yang, William R. Wilcox   +12 more
doaj   +2 more sources

Reducing agalsidase beta infusion time in Fabry patients: low incidence of antibody formation and infusion-associated reactions in an Italian multicenter study [PDF]

Orphanet Journal of Rare Diseases
Background Fabry disease is a rare progressive X-linked lysosomal storage disease caused by mutations in the GLA gene that encodes α-galactosidase A. Agalsidase beta is a recombinant enzyme replacement therapy authorized in Europe at a standard dose of 1.
Renzo Mignani, Claudio Americo, Filippo Aucella, Yuri Battaglia, Vittoria Cianci, Annamaria Sapuppo, Chiara Lanzillo, Fabio Pennacchiotti, Luciano Tartaglia, Giacomo Marchi, Federico Pieruzzi   +10 more
doaj   +2 more sources

Sex-based antibody subclass maturation drives direct enzymatic inhibition in fabry disease patients receiving enzyme replacement therapy [PDF]

Frontiers in Molecular Biosciences
BackgroundEnzyme replacement therapy (ERT) for Fabry disease can elicit anti-drug antibodies (ADAs) that may diminish efficacy and limit clinical benefit.ObjectivesTo develop and apply a multiplexed proteomic assay for quantitative, subclass-specific ADA
Tomas Baldwin, Hibba Kurdi, Ivan Doykov, Francesca Robertson, Stefania Rosmini, Sabrina Nordin, Sabrina Nordin, Joao Augusto, Rebecca Kozor, Rebecca Kozor, Julien Baruteau, Julien Baruteau, James Davison, David Moreno-Martinez, Uma Ramaswami, Ravi Vijapurapu, Tarekegn Geberhiwot, Rick Steeds, James Moon, James Moon, Derralynn Hughes, Derralynn Hughes, Kevin Mills, Wendy E. Heywood   +23 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
BackgroundTwo 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

A Fabry Disease Patient Who Developed Hypersensitivity Reaction against Agalsidase Beta following COVID-19 Infection. [PDF]

Nephron, 2023
Fabry disease (FD) is a rare, X-linked inherited lysosomal storage disorder, characterized by the accumulation of globotriaosylceramide (Gb3) due to the deficiency or absence of alpha-galactosidase A.
Sonmez O, Ozcan SG, Trabulus S, Seyahi N.   +3 more
europepmc   +2 more sources