Title:
USE OF AN AMINOGLYCOSIDE FOR NONSENSE MUTATION SUPPRESSION AND THE TREATMENT OF DISEASE
Document Type and Number:
WIPO Patent Application WO/2016/196927
Kind Code:
A8
Abstract:
Methods of using and pharmaceutical compositions comprising (2R,3S,4R,5R,6S)-5- amino-6-(((lR,2S,3S,4R,6S)-4,6-diamino-3-(((2R,3R,4R,5R)-3,5-dihydroxy-5-methyl-4-(methylamino)tetrahydro-2H-pyran-2-yl)oxy)-2-hydroxycyclohexyl)oxy)-2- (hydroxymethyl)tetrahydro-2H-pyran-3,4-diol are disclosed, including methods of treating or preventing a nonsense mutation mediated disease associated with premature translation termination or a premature stop codon resulting from a germline or somatic nonsense mutation.
Inventors:
BAIAZITOV RAMIL Y (US)
Application Number:
PCT/US2016/035709
Publication Date:
November 23, 2017
Filing Date:
June 03, 2016
Export Citation:
Assignee:
PTC THERAPEUTICS INC (US)
International Classes:
C07H15/23
Attorney, Agent or Firm:
BRUNER, Michael, J. et al. (US)
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