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US 2019/0194656 A1Published application
RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Duchenne and Becker Muscular Dystrophy
AssigneeBioMarin Technologies B.V.
InventorsPeter Christian de Visser, Judith Christina Theodora van Deutekom
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Abstract
The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.
Claims4
1-18. (canceled)
A 2′-O-methyl phosphorothioate oligonucleotide of 25 bases in length, having the base sequence GUUGX1X1UX1X1GGUUX1UGAAGGUGUUX1 (SEQ ID NO:
- 92), wherein each X1 is 5-methyl-cytosine, or a pharmaceutically acceptable salt thereof.
Dependencies are inferred from the claim text.