| 摘要 |
The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA. |
| 主权项 |
1. A compound of Formula 1-A-10 or 1-A-11:wherein:
n is 0, 1, or 2; R1 is carboxy, cyano, or a carbonyl group which is optionally substituted with a C1-C4 alkoxy group; R2 is absent or a nitro; R is hydrogen; a —Ra group; or two R groups, where R may also include an oxy group, together with the phenyl to which they are attached form a ring structure selected from RR; wherein: RR is a nine to ten membered bicyclic ring structure optionally substituted with one or more halogens, C1-C4 alkyl groups, C1-C4 haloalkyl groups, C1-C4 alkoxy groups, oxo groups, or C1-C4 haloalkoxy groups; Ra is selected from the group consisting of: a hydroxy group; a halogen; a C1-C4 alkyl which is substituted with one or more independently selected halogen or hydroxy groups; a C1-C4 alkoxy which is substituted with one or more independently selected halogen or phenyl groups; a C4-C8 cycloalkyl which is optionally substituted with one or more independently selected C1-C4 alkyl groups; an —Rb group; a —O—Rb group; a four to six-membered heterocycle which is optionally substituted with one or more independently selected C1-C4 alkyl, oxo, or —Rb groups; a nine to ten membered heterocycle having two ring structures; a carbonyl which is optionally substituted with a hydroxy, a C1-C4 alkyl, or a C1-C4 alkoxy group; a carbamoyl which is optionally substituted with one or two C1-C4 alkyl groups; a nitro group; a thio which is optionally substituted with a hydroxy, a C1-C4 alkyl, or —Rb group; a sulfonyl which is optionally substituted with a hydroxy, a C1-C4 alkyl, or —Rb group; or an amino which is optionally substituted with one or two independently selected C1-C4 alkyl, sulfonyl, or carbonyl groups, wherein the aminosulfonyl group is optionally substituted with a hydroxy, a C1-C4 alkyl, or —Rb group and wherein the aminocarbonyl group is optionally substituted with a C1-C4 alkyl, a C1-C4 haloalkyl, a benzoxy, or an amino group which is optionally substituted with an —Rb group; wherein —Rb is a C6-C8 aryl which is optionally substituted with one or more of the following: a hydroxy, a halogen, a C1-C4 alkyl group, a C1-C4 haloalkyl group, a C1-C4 alkoxy group, or an amino group which is optionally substituted with one or more C1-C4 alkyl groups; or a pharmaceutically acceptable salt thereof. |
