Methods, assays and compositions for treating...

Details Methods, assays and compositions for treating... Methods, assays and compositions for treating...

A - Human Necessities

61

K

A61K 31/16 (2006.01) A61K 31/07 (2006.01)

Patent

CA 2584845

Described herein are methods and compositions for treating certain retinol- related diseases and conditions by modulation of transthyretin (TTR) and retinol binding protein (RBP) availability in the subject. For example, the methods and compositions provide for therapeutic agents for the treatment and/or prevention of age-related macular degeneration and/or dystrophies, metabolic disorders, idiopathic intracranial hypertension, hyperostosis, and protein misfolding and aggregation diseases. The compositions disclosed may be used as single agent therapy or in combination with other agents or therapies. In addition, described herein are methods and assays for selecting appropriate agents that can modulate the TTR and RBP availability in a subject.

L'invention concerne des procédés et des compositions permettant de traiter certains états pathologiques et maladies liés au rétinol par la modulation de la disponibilité de la transthyrétine (TTR) et de la protéine de fixation du rétinol (RBP) chez un sujet. Les procédés et compositions permettent par exemple de traiter et/ou de prévenir la dégénérescence maculaire liée à l'âge et/ou des dystrophies, les troubles métaboliques, l'hypertension intracrânienne idiopathique, l'hyperostose et les maladies dues à un mauvais repliement et à l'agglomération de protéines. Les compositions décrites s'utilisent comme traitement unique ou en combinaison avec d'autres agents ou thérapies. L'invention concerne de plus des procédés et des dosages permettant de sélectionner des agents appropriés pouvant moduler la disponibilité de la TTR et de la RBP chez un sujet.

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