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US 7,465,738 B2Granted
Compounds useful as promoters of SMN2
AssigneeVertex Pharmaceuticals Incorporated
InventorsJill Jarecki, Xiaocun Chen, Dennis James Hurley, Lewis R. Makings, Mark T. Miller, Brian Pollok, Jeffrey H. Stack, Michael A. Whitney
Life of this patent
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Abstract
The present invention relates to compounds useful as promoters of the SMN2 gene. The invention also provides pharmaceutically acceptable compositions comprising said compounds and methods of using the compositions in the treatment of Spinal Muscular Atrophy.
Claims10
A method of treating or ameliorating one or more symptoms produced by Spinal Muscular Atrophy in a patient, comprising the step of administering to said patient a therapeutically effective amount of a compound of formula I:
- or a pharmaceutically acceptable salt thereof, wherein:
- X is O or S;
- R1 is a C1-6 aliphatic group, C6-10 aryl ring, heteroaryl ring having 5-10 ring atoms, or heterocyclyl ring having 3-10 ring atoms, each optionally substituted with 0-5 TR3;
- wherein each TR3 is independently halogen, CN, NO2, or an optionally substituted C1-4 alkyl, aryl, aralkyl, —N(R′)2, —CH2N(R′)—OR′, —Ch2OR′, —SR′, —CH2SR′,—COOR′,—NRCOR′,—CON(R′)2, or —S(O)2N(R′)2;
- each R2 is independently halogen, CN, NO2, or an optionally substituted C1-4 alkyl, aryl, aralkyl, —N(R′)3, —CH2N(R′)2, —OR′, CH2OR′, —SR′, CH2SR′, —COOR′, NRCOR′, —CON(R′)2, or —S(O)2N(R′)2;
- R′ is hydrogen or an optionally substituted C1-6 aliphatic group, an optionally substituted C6-10 aryl ring, an optionally substituted heteroaryl ring having 5-10 ring atoms, or an optionally substituted heterocyclyl ring having 3-10 ring atoms;
- and
- y is 0, 1, 2 or 3.
5 more elements
Dependencies are inferred from the claim text.