METHODS FOR TREATING SPINAL MUSCULAR ATROPHY

1 .- 21 . (canceled)

22 . A method for producing a stabilized SMNΔ7Ex7 protein in a human subject in need thereof, comprising administering to the human subject an effective amount of a compound of Formula (I) having the structure:

or a pharmaceutically acceptable salt, racemate, enantiomer or tautomer thereof, wherein,

W is C(O), C(S), and CH 2 ;

B is CH 2 or CH(C n H 2n+1 ), wherein n is an integer from 1 to 8;

R 1 and R 2 are independently selected from the group consisting of H and C 1 -C 3 alkyl, or R 1 and R 2 may be taken together with the carbon atom to which they are attached to form a C 3 -C 6 cycloalkyl ring or a carbonyl group;

R 3 is selected from the group consisting of H, halogen, C 1 -C 4 alkyl, C 1 -C 4 alkoxy, C 1 -C 4 haloalkyl, CN, NO 2 , heteroaryl, and phenyl optionally substituted with any combination of one to five halogen, NO 2 , CN, C 1 -C 4 alkyl, C 1 -C 4 haloalkyl, or C 1 -C 4 alkoxy substituents;

R 4 , R 5 , R 6 and R 7 are independently selected from the group consisting of H, hydroxyl, halogen, CN, NO 2 , sulfonamide, C 1 -C 8 alkyl, C 3 -C 6 cycloalkyl, cycloalkyloxy, C 1 -C 6 alkoxy, C 1 -C 6 haloalkoxy, C 1 -C 4 haloalkyl, C 2 -C 8 alkenyl, amino, C 1 -C 4 alkylamino, C 1 -C 4 dialkylamino, C 3 -C 6 cycloalkylamino, morpholinyl, heteroaryl, arylamino, arylalkylamino, phenyl, C(O)R′, NR′(COR″), NR′SO 2 R″ and NR′(CONR″R′″), wherein R′, R″ and R′″ are independently H, C 1 -C 6 alkyl, phenyl, or substituted phenyl, and wherein C 1 -C 8 alkyl is optionally substituted with one or more substituents selected from the group consisting of C 1 -C 4 alkoxy, C 1 -C 4 haloalkyl, C 1 -C 6 dialkylamino, C 1 -C 6 alkylamino, cycloalkylamino, and morpholinyl, and the phenyl is optionally substituted with one or more substituents selected from the group consisting of halogen, NO 2 , CN, C 1 -C 4 alkyl, C 1 -C 4 haloalkyl, and C 1 -C 4 alkoxy, or R 4 and R 5 , R 5 and R 6 , or R 6 and R 7 , taken together with the carbon to which they are attached, form a ring;

X is selected from the group consisting of H; CN; C(O)OR 8 , wherein R 8 is H or C 1 -C 8 alkyl, and C 1 -C 8 alkyl optionally is substituted with one or more substituents selected from the group consisting of C 1 -C 4 alkoxy, C 1 -C 4 haloalkyl, C 1 -C 6 dialkylamino, C 1 -C 6 alkylamino, cycloalkylamino, phenyl, and morpholinyl; C(O)NR 9 R 10 or CH 2 NR 9 R 10 , wherein R 9 and R 10 are independently selected from the group consisting of H and C 1 -C 6 alkyl, or R 9 and R 10 together with the nitrogen to which they are attached form a heterocyclyl ring; CH 2 OR 11 , wherein R 11 is H, C 1 -C 8 alkyl, or C 3 -C 6 cycloalkyl, wherein C 1 -C 8 alkyl is optionally substituted with one or more substituents selected from the group consisting of C 1 -C 4 alkoxy, C 1 -C 4 haloalkyl, C 1 -C 6 dialkylamino, C 1 -C 6 alkylamino, cycloalkylamino, and morpholinyl; CH 2 Z, wherein Z is halogen; C(O)NHOH; C(O)NHCN; C(O)N(R 1 )SO 2 R 13 , wherein R 13 is C 1 -C 4 alkyl, phenyl, or substituted phenyl; C 1 -C 8 alkyl, optionally substituted with one or more substituents selected from the group consisting of C 1 -C 4 alkoxy, C 1 -C 4 haloalkyl, C 1 -C 6 dialkylamino, and C 1 -C 6 alkylamino; and C 2 -C 8 alkenyl, optionally substituted with one or more substituents selected from the group consisting of C 1 -C 4 alkoxy, C 1 -C 4 haloalkyl, C 1 -C 6 dialkylamino, and C 1 -C 6 alkylamino.

23 . The method of claim 22 , wherein the stabilized SMNΔ7Ex7 protein comprises the amino acid sequence of SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, or SEQ ID NO: 5.

24 .- 31 . (canceled)

32 . The method of claim 22 , wherein the compound is 2-(4-isopropylphenyl)-6-methoxyisoindolin-1-one.

33 . The method of claim 22 , wherein:

W is C(O);

B is CH 2 ;

R 1 and R 2 are independently C 1 -C 3 alkyl;

R 3 is H;

R 4 , R 5 , R 6 and R 6 are independently selected from the group consisting of H and C 1 -C 6 alkoxy; and

X is H.

34 . The method of claim 33 , wherein the compound has the structure:

or a pharmaceutically acceptable salt, racemate, enantiomer or tautomer thereof.