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US 10,420,753 B2Granted
Combination therapies for treatment of spinal muscular atrophy
AssigneeIndiana University Research and Technology Corporation
InventorsElliot J. Androphy, Kevin Hodgetts, Alyssa Nicole Calder
Life of this patent
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Abstract
Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.
Claims18
A compound having the formula of formula II:
- wherein X is selected from the group consisting of carbon or nitrogen;
- Y is selected from the group consisting of carbon or nitrogen;
- R2 is selected from unsubstituted or substituted phenyl, unsubstituted or substituted heteroaryl, wherein the unsubstituted heteroaryl is selected from the group consisting of a 6-membered heteroaryl and a 5-membered heteroaryl, and wherein when R2 is a substituted phenyl or substituted heteroaryl, the substitution is selected from the group consisting of hydrogen, halogen, lower alkyl and combinations thereof;
- and
- wherein at least one of X or Y is nitrogen.
2 more elements
Dependencies are inferred from the claim text.