A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/445 (2006.01) A61K 48/00 (2006.01) A61P 21/00 (2006.01) C12N 9/26 (2006.01)
Patent
CA 2612538
The present invention provides a method for increasing the activity of a mutant or wild-type .alpha.-glucosidase enzyme in vitro and in vivo by contacting the enzyme with a specific pharmacological chaperone which is a derivative of 1-deoxynojirimycin. The invention also provides a method for the treatment of Pompe disease by administration of chaperone small molecule compound which is a derivative of 1-deoxynojirimycin. The 1-deoxynojirimycin derivative is substituted at the N or Cl position. Combination therapy with replacement .alpha.-glucosidase gene or enzyme is also provided.
L'invention concerne une méthode pour augmenter l'activité d'une enzyme .alpha.-glucosidase de type mutant ou sauvage, in vitro et in vivo, par la mise en contact de cette enzyme avec un chaperon pharmacologique spécifique qui est un dérivé de 1-désoxynojirimycine. L'invention concerne également une méthode pour traiter la maladie de Pompe consistant à administrer un composé chaperon se présentant sous la forme d'une petite molécule qui est un dérivé de 1-désoxynojirimycine. Le dérivé de 1-désoxynojirimycine est substitué en position N ou C1. Une polythérapie impliquant un gène ou une enzyme .alpha.-glucosidase de remplacement est également décrite.
Boyd Robert
Mugrage Benjamin
Sheth Kamlesh
Zhu Xiaoxiang
Amicus Therapeutics Inc.
Bereskin & Parr Llp/s.e.n.c.r.l.,s.r.l.
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