A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 47/48 (2006.01) A61K 38/43 (2006.01) A61K 47/42 (2006.01) A61M 25/00 (2006.01)
Patent
CA 2522609
The present invention is directed to methods and systems for the treatment of inborn genetic errors or other defects that cause deficiencies of active enzymes of proteins within the cells of the central nervous system. Such methods and systems generally comprise an implantable catheter system designed for the chronic delivery of specially formulated proteins to intrathecal, intracerebroventricular, and/or intraparenchymal regions of the central nervous system. The invention has application in the neuropathic aspects of the broad category of lysosomal storage diseases. These genetic based diseases are the result of insufficient enzyme activity to catabolize specific substances, which thereby accumulate in the cellular lysosomes.
La présente invention concerne des méthodes et des systèmes de traitement d'erreurs génétiques congénitales ou autres défauts qui provoquent des déficiences enzymatiques de protéines dans les cellules du système nerveux central. Ces méthodes et systèmes font généralement appel à un système de cathéter implantable pour l'administration continue de protéines spécialement formulées pour des régions intrathécales, intraacérébroventriculaires et/ou intraparenchymateuses du système nerveux central. L'invention s'applique aux aspects nevropathiques d'une vaste catégories de maladies lysosomales. Ces maladies d'origine génétique sont dues à une activité enzymatique qui est insuffisante pour catabolyser des substances spécifiques qui s'accumulent ipso facto dans les lysosomes cellulaires.
Kaemmerer William F.
Keimel John G.
Medtronic Inc.
Smart & Biggar
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