Matthew G. Woll,Guangming Chen,Soongyu Choi,Amal Dakka,Song Huang,Gary Mitchell Karp,Chang-Sun Lee,Chunshi Li,Jana Narasimhan,Nikolai Naryshkin,Sergey Paushkin,Hongyan Qi,Anthony A. Turpoff,Marla L. W
申请号：
US15695299
公开号：
USRE047689E1
申请日:
2017.09.05
申请国别(地区):
US
年份:
2019
代理人:
摘要：
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.
