Intraventricular enzyme delivery for lysosomal storage diseases

Details Intraventricular enzyme delivery for lysosomal storage diseases Intraventricular enzyme delivery for lysosomal storage diseases

A - Human Necessities

61

K

A61K 38/46 (2006.01)

Patent

CA 2636991

Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood- brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.

La présente invention concerne des maladies de stockage des lysosomes pouvant être traitées avec succès par une administration intraventriculaire de l~enzyme qui est étiologiquement insuffisante dans la maladie. L~administration peut être effectuée lentement pour obtenir un effet maximal. Il est surprenant de constater que des effets sont visibles sur les deux côtés de la barrière hématoencéphalique, faisant de ce procédé un moyen d~administration idéal pour des maladies de stockage des lysosomes affectant à la fois le cerveau et des organes viscéraux.

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