Use of an aminoglycoside for nonsense mutation suppression and the treatment of disease
AssigneePTC Therapeutics, Inc.
InventorRamil Y. Baiazitov
Life of this patent
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Abstract
Methods of using and pharmaceutical compositions comprising (2R,3S,4R,5R,6S)-5-amino-6-(((1R,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.
Claims10
A method for treating a disease caused by premature translation termination, a nonsense mutation or a premature stop codon, for the purpose of minimizing the spread or worsening of said disease, comprising administering to a patient having said disease an effective amount of a compound having the structure of Compound 1:
- having the name (2R,3S,4R,5R,6S)-5-amino-6-(((1R,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, or a pharmaceutically acceptable salt or hydrate thereof;
- wherein, the compound is a purified stereoisomer.
Dependencies are inferred from the claim text.