Is a
Patent attributes
Patent Jurisdiction
Patent Number
Patent Inventor Names
Jana Narasimhan0
Marla L. Weetall0
Matthew G. Woll0
Nanjing Zhang0
Nikolai Naryshkin0
Sergey Paushkin0
Song Huang0
Soongyu Choi0
...
Date of Patent
April 11, 2017
0Patent Application Number
143692940
Date Filed
December 28, 2012
0Patent Citations Received
Patent Primary Examiner
Patent abstract
Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.
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