C - Chemistry – Metallurgy – 07 – D
Patent
C - Chemistry, Metallurgy
07
D
C07D 263/57 (2006.01) A61K 31/423 (2006.01) G01N 33/68 (2006.01)
Patent
CA 2510455
Kinetic stabilization of the native state of transthyretin is an effective mechanism for preventing protein misfolding. Because transthyretin misfolding plays an important role in transthyretin amyloid diseases, inhibiting such misfolding can be used as an effective treatment or prophylaxis for such diseases. Treatment methods, screening methods, as well as specific transthyretin stabilizing compounds are disclosed.
La stabilisation cinétique de l'état natif de la transthyrétine est un mécanisme efficace de prévention du mauvais repliement des protéines. Etant donné que le mauvais repliement de la transthyrétine joue un rôle important dans les maladies amyloïdes associées à la transthyrétine, l'inhibition de ce mauvais repliement peut constituer un traitement ou une prophylaxie efficace pour ce type de maladies. L'invention décrit des méthodes de traitement, des méthodes de criblage et des composés spécifiques stabilisant la transthyrétine.
Kelly Jeffery W.
Powers Evan T.
Razavi Hossein
Sekijima Yoshiki
Osler Hoskin & Harcourt Llp
The Scripps Research Institute
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