| 发明名称 |
COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY |
| 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. |
| 申请公布号 |
KR20140128310(A) |
申请公布日期 |
2014.11.05 |
| 申请号 |
KR20147021262 |
申请日期 |
2012.12.28 |
| 申请人 |
PTC THERAPEUTICS, INC.;F. HOFFMANN-LA ROCHE AG |
发明人 |
WOLL MATTHEW 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 ANTHONY A.;WEETALL MARLA L.;WELCH ELLEN;YANG TIANLE;ZHANG NANJING;ZHANG XIAOYAN;ZHAO XIN;PINARD EMMANUEL;RATNI HASANE |
| 分类号 |
C07D413/14;A61K31/353;A61P21/00;C07D405/04;C07D417/14 |
主分类号 |
C07D413/14 |
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