Results 81 to 90 of about 744,838 (140)

Pegunigalsidase alfa: a novel, pegylated recombinant alpha-galactosidase enzyme for the treatment of Fabry disease

Frontiers in Genetics
Fabry disease, a rare X-linked genetic disorder, results from pathogenic variants in GLA, leading to deficient lysosomal α-galactosidase A enzyme activity and multi-organ manifestations. Since 2001, enzyme replacement therapy (ERT), using agalsidase alfa
Dominique P. Germain, Dominique P. Germain, Ales Linhart   +2 more
doaj   +1 more source

FCA-2022-0047 - German - Plain language summary of a study looking at heart muscle thickness and kidney function in women with Fabry disease who received agalsidase beta treatment

, 2023
What is Fabry disease & what is this study about? Fabry disease is a rare genetic condition that affects many different cells and organs in the body. People have Fabry disease when they inherit a GLA gene containing an error from one or both of their ...
Ulla Feldt-Rasmussen (317604), Alberto Ortiz (108398), Christoph Wanner (156898)   +2 more
core   +1 more source

Anti-alpha-galactosidase A antibody response to agalsidase beta treatment: Data from the Fabry Registry

, 2012
Agalsidase beta, a form of recombinant human alpha-galactosidase A (alpha GAL), is approved for use as enzyme replacement therapy (ERT) for Fabry disease.
Germain, Dominique P, Warnock, David G, Linthorst, Gabor E., Richards, Susan M, Wilcox, William R., Linthorst, Gabor E, Germain, Dominique P., Beitner-Johnson, Dana, Kingma, Wytske, Cole, J. Alexander, Richards, Susan M., Cizmarik, Marta, Feldt-Rasmussen, Ulla, Wilcox, William R, Cole, J Alexander, Warnock, David G., Waldek, Stephen   +16 more
core   +1 more source

The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts

Molecular Genetics and Metabolism Reports, 2019
Background: Enzyme replacement therapy (ERT) with recombinant human α-galactosidase has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the USA.
Dominique P. Germain, Perry M. Elliott, Bruno Falissard, Victor V. Fomin, Max J. Hilz, Ana Jovanovic, Ilkka Kantola, Aleš Linhart, Renzo Mignani, Mehdi Namdar, Albina Nowak, João-Paulo Oliveira, Maurizio Pieroni, Miguel Viana-Baptista, Christoph Wanner, Marco Spada   +15 more
doaj   +1 more source

Safety and Efficacy of Enzyme Replacement Therapy with Agalsidase Beta: An International, Open-label Study in Pediatric Patients with Fabry Disease

, 2008
Objective: To evaluate the safety and explore the efficacy of enzyme replacement therapy with agalsidase beta (recombinant human α-galactosidase A; Fabrazyme [Genzyme Corporation, Cambridge, MA]) in pediatric patients with Fabry disease, a genetic ...
Wraith, J. Edmond, Lien, Y. Howard, Guffon, Nathalie, Tylki-Szymanska, Anna, Tsimaratos, Michel, Germain, Dominique P., Vellodi, Ashok   +6 more
core   +1 more source

Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease

Biologics: Targets & Therapy, 2008
Fernando C Fervenza1, Roser Torra2, David G Warnock31Division of Nephrology and Hypertension, Mayo Clinic College of Medicine, Rochester, MN, USA; 2Department of Nephrology, Fundació Puigvert, Barcelona, Spain; 3Division of Nephrology ...
Fernando C Fervenza, Roser Torra, David G Warnock   +2 more
doaj  

Phi Beta Kappa Minutes, December 5, 1776-January 6,1781 [PDF]

Ledger of the Phi Beta Kappa minutes of meetings from December 5, 1776-January 6, 1781. These are the earliest meetings of Phi Beta Kappa.
Phi Beta Kappa
core  

Agalsidase beta therapy for advanced fabry disease: A randomized trial

Fabry disease is a rare, X-linked lysosomal storage disorder that can cause early death from renal, cardiac, and cerebrovascular involvement. In this double-blind multicenter trial, 82 adults with kidney dysfunction from Fabry disease were randomly ...
-
core  

Phi Beta Kappa: The Key Reporter Winter 2012-2013

, 2012
Announcement of the Beta of Nebraska chapter installation at Creighton University, p.
Phi Beta Kappa Society
core  

A phase II, multicenter, open-label trial to evaluate the safety and efficacy of ISU303 (Agalsidase beta) in patients with Fabry disease. [PDF]

Medicine (Baltimore), 2022
Hwang S, Lee BH, Kim WS, Kim DS, Cheon CK, Lee CH, Choi Y, Choi JH, Kim JH, Yoo HW.   +9 more
europepmc   +1 more source