| 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 |
