Chaperone-based therapy for niemann-pick disease

Details Chaperone-based therapy for niemann-pick disease Chaperone-based therapy for niemann-pick disease

A - Human Necessities

61

K

A61K 31/74 (2006.01)

Patent

CA 2546863

The present invention provides a method for treating individuals affected with the acid sphingomyelinase-deficient forms of Niemann-Pick disease (i.e., Type A or Type B Niemann-Pick) by administering small molecules as specific molecular "chaperones" for the deficient acid sphingomyelinase (ASM) enzyme associated with the disease. The molecules are ceramide, sphingomyelin, or phosphonucleotide analogues.

La présente invention concerne un procédé de traitement d'individus souffrant des formes déficientes en sphingomyélinase acide de la maladie de Niemann-Pick (notamment Niemann-Pick de type A ou de type B) par l'administration de petites molécules comme les <= chaperones" moléculaires spécifiques de l'enzyme déficient en sphingomyelinase acide (ASM) associé à la maladie. Les molécules sont de la céramide, la sphingomyéline ou les analogues de phosphonucléotide.

Affiliated with

Also associated with

Rating

Chaperone-based therapy for niemann-pick disease does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Chaperone-based therapy for niemann-pick disease, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Chaperone-based therapy for niemann-pick disease will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFCA-PAI-O-1970260