A randomised, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of three dosing schedules of agalsidase alfa enzyme replacement …
DA Hughes, PB Deegan, A Milligan, N Wright… - Molecular genetics and …, 2013 - Elsevier
Anecdotal reports suggest that the currently approved dosing interval of agalsidase alfa (0.2
mg/kg/2weeks) for Fabry disease treatment is too long. This randomised, double-blind,
placebo-controlled, crossover study investigated three altered dosing intervals. 18 Fabry
patients received three agalsidase alfa dosing schedules, each for four weeks (A: 0.2
mg/kg∗ 2weeks, B: 0.1 mg/kg/week, C: 0.2 mg/kg/week). Health state, pain levels, sweat
volume and latency and plasma and urinary globotriaosylceramide levels were recorded …
mg/kg/2weeks) for Fabry disease treatment is too long. This randomised, double-blind,
placebo-controlled, crossover study investigated three altered dosing intervals. 18 Fabry
patients received three agalsidase alfa dosing schedules, each for four weeks (A: 0.2
mg/kg∗ 2weeks, B: 0.1 mg/kg/week, C: 0.2 mg/kg/week). Health state, pain levels, sweat
volume and latency and plasma and urinary globotriaosylceramide levels were recorded …
