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Patent Searching and Data


Title:
METHODS AND COMPOUNDS FOR STIMULATING READ-THROUGH OF PREMATURE TERMINATION CODONS
Document Type and Number:
WIPO Patent Application WO/2017/181193
Kind Code:
A3
Abstract:
The present disclosure provides compositions and methods for treating and/preventing diseases and conditions associated with premature termination mutations. The methods disclosed herein comprise the step of administering to the subject a therapeutically effective dose of a compound described herein that induces read-through of the premature termination codon. The methods of the present disclosure may further comprise treating the subject with enzyme replacement therapy wherein the enzyme replacement therapy is selected for the disease or condition to be treated. Furthermore, the present disclosure provides method of pharmacologically suppressing premature termination codons in a subject in need of such suppression.

Inventors:
ROWE STEVEN (US)
BEDWELL DAVID M (US)
MUTYAM VENKATESHWAR (US)
DU MING (US)
KEELING KIM M (US)
Application Number:
US2017/027982
Publication Date:
November 23, 2017
Filing Date:
April 17, 2017
Export Citation:
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Assignee:
UAB RES FOUND (US)
International Classes:
A61K31/035; A61K47/06; A61K47/26; C07H19/00; C07H19/01; C12Q3/00
Foreign References:
US20150080431A12015-03-19
US20050233327A12005-10-20
US20140135296A12014-05-15
EP2149378B12014-08-06
Other References:
MUTYAM ET AL.: "Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations", AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, vol. 194, no. 9, 1 November 2016 (2016-11-01), XP055440109, Retrieved from the Internet [retrieved on 20170920]
Attorney, Agent or Firm:
PETERSON, Thomas G. et al. (Birmingham, Alabama, US)
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