| abstract |
The present invention provides a glucocerebrosidase composition, such as velaglucerase alfa, and isofagomine, which are present in a molar ratio of at least about 1: 2.5. The invention also relates to a use of the composition for treating a disorder related to a dysfunction of a glucocerebrosidase pathway. The disorder may be a lysosomal disease, such as Gaucher's disease, Fabry's disease, Pompe's disease, mucopolysaccharidosis, or multi-system atrophy. The disorder may also be a neurodegenerative disorder, such as Parkinson's disease, Alzheimer's disease or Lewy body dementia. The composition may comprise 0.5 to 5.0 mg / kg of glucocerebrosidase and isofagomine in molar excess of at least about 3-fold relative to glucocerebrosidase. The composition can be administered intravenously or subcutaneously. |