Ptc Therapeutics, Inc.

F. Hoffmann-la Roche Ag

Song Xiao Huang

Gary Mitchell Karp

Guangming Chen

Xiaoyan Zhang

Nanjing Zhang

Anthony A Turpoff

Chunshi Li

Emmanuel Pinard

Hasane Ratni

Ellen Welch

Nikolai A Naryshkin

Soongyu Choi

Hongyan Qi

Marla L Weetall

Jana Narasimhan

Amal Dakka

Matthew G Woll

Xin Zhao

Chang-Sun Lee

Tianle Yang

Sergey Paushkin

F George Njoroge

Malcolm MacCoss

James Jan Takasugi

Steven David Paget

Hongyu Ren

Ronggang Liu

Ramesh Kakarla

Amin Nomeir

Sean Wesley Smith

Jin Zhu

Christie Morrill

Yalei Liu

William Joseph Lennox

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.

Compounds for treating spinal muscular atrophy

The present invention is directed to compounds and forms and pharmaceutical compositions thereof useful for treating a viral infection, or for affecting viral activity by modulating viral replication.

Indole derivatives and methods for antiviral treatment

Growing community of inventors

New Providence, NJ, United States of America

Song Xiao Huang