Frequently asked questions


“Vimizim is changing Sam’s life. This is clinically proven, but I don’t need clinical evidence. I can see it every day.”


“Kamal’s only wish for Father Christmas is ‘to be a normal boy’, and by having Vimizim it gives him the chance to be as normal as can be.”


“…just being able to get on with life and enjoy my leisure time as everyone else does, without constantly feeling exhausted, has had a very big impact on my quality of life.”


“We can’t even bear to think about the fact that it may not be funded. It would feel like we had given Gracie the best chance, to have it snatched away from her."


"I have noticed my body really craves the enzyme replacement treatment, I’m very scared about it discontinuing and going back to that dark place.”