WOLL, MATTHEW G. G,CHEN, GUANGMING,CHOI, SOONGYU,DAKKA, AMAL,HUANG, SONG,KARP, GARY MITCHELL,LEE, CHANG-SUN,LI, CHUNSHI,NARASIMHAN, JANA,NARYSHKIN, NIKOLAI,PAUSHKIN, SERGEY,QI, HONGYAN,TURPOFF, ANTHON,馬修‧,‧沃爾,陳光明,崔珣奎,阿邁爾‧達卡,松‧黃,加里‧米切爾‧卡普,長勳‧李,春世‧李,NARYSHKIN, NIKOL
申请号：
HK15102568.1
公开号：
HK1202059A
申请日:
2015.03.13
申请国别(地区):
HK
年份:
2015
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.
