Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT).
The shortage of enzyme for treatment of Fabry disease has caused anxiety among patients, physicians and governments. Following a request from the European Medicines Agency, consensus was reached on the temporary prioritization of patients for treatment based on disease severity and potential reversibility. Advice on follow-up of patients was agreed upon. This consensus is proposed to support the temporary guidelines issued throughout the period of ERT shortage, which will most likely last until April 2011.
Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Center, Amsterdam, The Netherlands. g.e.linthorst@amc.uva.nl
Gabor E
GE
Linthorst
Dominique P
DP
Germain
Carla E M
CE
Hollak
Derralynn
D
Hughes
Arndt
A
Rolfs
Christoph
C
Wanner
Atul
A
Mehta
D056947:Enzyme Replacement Therapy; D006801:Humans; D007527:Isoenzymes / Q000008:administration & dosage / Q000600:supply & distribution*; D000519:alpha-Galactosidase / Q000008:administration & dosage / Q000600:supply & distribution*
D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta