PTC Therapeutics, Inc.
Use of an aminoglycoside for nonsense mutation suppression and the treatment of disease

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,5- R)-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.