公开(公告)号：EP2797592A4

公开(公告)日：2016-01-13

申请号：EP12862080

申请日：2012-12-28

申请人： PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

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

IPC分类号： A61K31/353 ,  A61P25/00 ,  C07D231/12 ,  C07D311/16 ,  C07D311/18 ,  C07D405/04 ,  C07D405/12 ,  C07D405/14 ,  C07D413/04 ,  C07D413/14 ,  C07D417/04 ,  C07D417/14 ,  C07D471/04 ,  C07D487/04 ,  C07D491/04 ,  C07D495/04 ,  C07D513/04 ,  C07D519/00

CPC分类号： C07D487/04 ,  C07D231/12 ,  C07D311/16 ,  C07D311/18 ,  C07D405/04 ,  C07D405/12 ,  C07D405/14 ,  C07D413/04 ,  C07D413/14 ,  C07D417/04 ,  C07D417/14 ,  C07D471/04 ,  C07D491/04 ,  C07D495/04 ,  C07D513/04 ,  C07D519/00

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.

公开(公告)号：EP2750678A4

公开(公告)日：2015-04-08

申请号：EP12826819

申请日：2012-08-29

申请人： PTC THERAPEUTICS INC

发明人： BRANSTROM ARTHUR ,  JOSYULA VARA PRASAD VENKATA NAGENDRA ,  ARNOLD MICHAEL ANDREW ,  GERASYUTO ALEKSEY I ,  KARP GARY ,  WANG JIASHI ,  CHEN GUANGMING ,  GINZBURG OLYA ,  HUANG SONG ,  PEDDI SRINIVASA ,  WOLL MATTHEW G ,  SMITH SEAN WESLEY ,  ZHANG XIAOYAN ,  ZHANG NANJING ,  TURPOFF ANTONY ALLAN ,  NARASIMHAN JANA

IPC分类号： A61K31/473 ,  C07D221/14 ,  C07D221/16 ,  C07D471/04 ,  C07D471/14 ,  C07D491/04 ,  C07D495/04

CPC分类号： C07D491/052 ,  A61K31/435 ,  A61K31/4353 ,  A61K31/436 ,  A61K31/437 ,  A61K31/438 ,  A61K31/473 ,  A61K31/496 ,  A61K31/5377 ,  A61K31/55 ,  A61K45/06 ,  C07D221/10 ,  C07D221/14 ,  C07D221/16 ,  C07D401/04 ,  C07D471/04 ,  C07D471/14 ,  C07D491/044 ,  C07D491/048 ,  C07D491/14 ,  C07D495/04 ,  C07D519/00 ,  Y02P20/582 ,  A61K2300/00

摘要： The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens.