Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain

BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by α-galactosidase A deficiency leading to renal, cardiac, cerebrovascular disease and premature death. Treatment with α-galactosidase A (enzyme replacement therapy, ERT) stabilises disease in some patients, but long term eff...

Full beskrivning

Sparad:
Bibliografiska uppgifter
Huvudupphovsmän: Rombach, Saskia M, Smid, Bouwien E, Bouwman, Machtelt G, Linthorst, Gabor E, Dijkgraaf, Marcel G W, Hollak, Carla E M
Materialtyp: Artigo
Språk:Inglês
Publicerad: BioMed Central 2013
Ämnen:
Research
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC3626869/
https://ncbi.nlm.nih.gov/pubmed/23531228
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-47
Taggar: Lägg till en tagg
Inga taggar, Lägg till första taggen!

Liknande verk