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

BACKGROUND: 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. An independent international initiative compared clinical and biochemical outcomes of the...

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Pubblicato in:J Med Genet
Autori principali: Arends, Maarten, Biegstraaten, Marieke, Wanner, Christoph, Sirrs, Sandra, Mehta, Atul, Elliott, Perry M, Oder, Daniel, Watkinson, Oliver T, Bichet, Daniel G, Khan, Aneal, Iwanochko, Mark, Vaz, Frédéric M, van Kuilenburg, André B P, West, Michael L, Hughes, Derralynn A, Hollak, Carla E M
Natura: Artigo
Lingua:Inglês
Pubblicazione: BMJ Publishing Group 2018
Soggetti:
Therapeutics
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5931248/
https://ncbi.nlm.nih.gov/pubmed/29437868
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/jmedgenet-2017-104863
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