A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.

@article{Connock2006ASR,
  title={A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.},
  author={Martin J. Connock and Ariadna Ju{\'a}rez-Garc{\'i}a and Edward Frew and Anton Mans and Janine Dretzke and Anne Fry-Smith and Dana Moore},
  journal={Health technology assessment},
  year={2006},
  volume={10 20},
  pages={iii-iv, ix-113}
}
OBJECTIVES To determine the clinical effectiveness and cost-effectiveness of the administration of intravenous enzyme replacement therapy (ERT) to symptomatic patients for the prevention of long-term damage and symptoms in Fabry's disease and in mucopolysaccharidosis type 1 (MPS1). DATA SOURCES Electronic databases from inception up to mid-2004. Contact… CONTINUE READING