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1. (WO2017181193) METHODS AND COMPOUNDS FOR STIMULATING READ-THROUGH OF PREMATURE TERMINATION CODONS

Pub. No.:    WO/2017/181193    International Application No.:    PCT/US2017/027982
Publication Date: Fri Oct 20 01:59:59 CEST 2017 International Filing Date: Tue Apr 18 01:59:59 CEST 2017
IPC: A61K 31/035
A61K 47/06
A61K 47/26
C07H 19/00
C07H 19/01
C12Q 3/00
Applicants: THE UAB RESEARCH FOUNDATION
Inventors: ROWE, Steven
BEDWELL, David M.
MUTYAM, Venkateshwar
DU, Ming
KEELING, Kim M.
Title: METHODS AND COMPOUNDS FOR STIMULATING READ-THROUGH OF PREMATURE TERMINATION CODONS
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.