Ptc Therapeutics, Inc.

F. Hoffmann-la Roche Ag

Bayer Pharmaceuticals Corporation

Bayer Healthcare LLC

Pct Therapeutics, Inc.

Soongyu Choi

Hongyan Qi

Young-Choon Moon

William Jospeh Lennox

Hasane Ratni

Gary Mitchell Karp

Marla L Weetall

Emmanuel Pinard

Ellen Welch

Nikolai A Naryshkin

Xiaoyan Zhang

Liangxian Cao

Nanjing Zhang

Jana Narasimhan

Amal Dakka

Matthew G Woll

Xin Zhao

Tianle Yang

Donald Thomas Corson

Nadarajan Tamilarasu

Seongwoo Hwang

Anthony A Turpoff

Rico C Lavoie

Luke Green

Roger B Clark

Xin Ma

Martin F Hentemann

Chang-Sun Lee

Joachim Rudolph

Louis-David Cantin

Zhonghua Zhang

Thomas W Davis

Roger Astbury Smith

Guangming Chen

Wai C Wong

Stephen J O'Connor

Sidney Xi Liang

Philip L Wickens

Chunshi Li

Sergey Paushkin

Dyuti Majumdar

Libing Chen

Christiana Akuche

Song Xiao Huang

Duck-Hyung Lee

Don Corson

Jeff Campbell

William Lennox

Neil Gregory Almstead

William Johnston Scott

Jeffrey Allen Campbell

Harold C Kluender

Gan Wang

Jill E Wood

Richard Gerald Wilde

David Gunn

Peter Seongwoo Hwang

Brian R Dixon

Langdon Miller

Steven David Paget

Peiying Liu

Jianmei Fan

Hongyu Ren

Nadiya Sydorenko

Derek B Lowe

Yamin Wang

Ning Miao Su

James H Cook, Ii

Christopher Robert Trotta

Harold C E Kluender

Ramil Y Baiazitov

Jeremy L Baryza

Catherine R Brennan

Wu Du

Zhengxian Gu

Ronggang Liu

Stephan-Nicholas Wirtz

Shihong Ying

Furahi Achebe

Brent L Podlogar

Harry H Miao

Hiroo Koyama

Paul P Ehrlich

Tamil Arasu

Anik&oacute; M Redman

Jason D Graci

Ian McAlexander

Anthony A Yurpoff

William Joseph Lennox

Thomas Davis

Maria L Weetall

Song Huang

Sidney X Liang

Provided herein are compounds of Formula (I): and forms thereof, wherein w, w, w, w, w, wand ware as defined herein, including compositions thereof and uses therewith for treating spinal muscular atrophy.

Compounds for treating spinal muscular atrophy

Provided herein are compounds of Formula (I): 

Amine substituted pyridine and pyrazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Substituted pyridine and pyrazine BMI-1 inhibitors

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.

Provided herein are compounds of Formula (I): and forms thereof, including compositions thereof and uses therewith for treating spinal muscular atrophy.

Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

Compounds, such as compounds of Formula (I), that selectively inhibit pathological production of human vascular endothelial growth factor (VEGF) and compositions comprising such Compounds are described. Compounds that inhibit viral replication or the production of viral RNA or DNA or viral protein and compositions comprising such Compounds are described. Also described are methods of reducing VEGF using such Compounds and methods for treating cancer and non-neoplastic conditions involving the administration of such Compounds. Further described are methods of inhibiting viral replication or the production of viral RNA or DNA or viral protein using such Compounds and methods for treating viral infections involving the administration of such Compounds. The Compounds may be administered as a single agent therapy or in combination with one or more additional therapies to a human in need of such treatments. 

Methods for treating cancer and non-neoplastic conditions

The present invention relates to methods, compounds, and compositions for inhibiting angiogenesis. More particularly, the present invention relates to methods, compounds, and compositions for inhibiting VEGF production.

Tetrahydrocarbazoles as active agents for inhibiting VEGF production by translational control

In accordance with the present invention, tetracyclic caboline derivatives that inhibit the expression of VEGF post-transcriptionally have been identified, and methods for their use provided. In one aspect of the invention, these compounds useful in the inhibition of VEGF production, in the inhibition of angiogenesis, and/or in the treatment of cancer, diabetic retinopathy or exudative macular degeneration are provided. In another aspect of the invention, methods are provided for the inhibition of VEGF production, the inhibition of angiogenesis, and/or the treatment of cancer, diabetic retinopathy or exudative macular degeneration using the compounds of the invention.

Tetra-cyclic carboline derivatives useful in the inhibition of angiogenesis

In accordance with the present invention, compounds that inhibit the expression of VEGF post-transcriptionally have been identified, and methods for their use provided. In one aspect of the invention, compounds useful in the inhibition of VEGF production, in the treatment of solid tumor cancer, and in reducing plasma and/or tumor VEGF levels, are provided. In another aspect of the invention, methods are provided for the inhibition of VEGF production, the treatment of cancer, and the reduction of plasma and/or tumor VEGF levels, using the compounds of the invention.

Carboline derivatives useful in the inhibition of angiogenesis

In accordance with the present invention, compounds that inhibit the expression of VEGF post-transcriptionally have been identified, and methods for their use provided. In one aspect of the invention, compounds useful in the inhibition of VEGF production, in the inhibition of angiogenesis, and/or in the treatment of cancer, diabetic retinopathy or exudative macular degeneration are provided. In another aspect of the invention, methods are provided for the inhibition of VEGF production, the inhibition of angiogenesis, and/or the treatment of cancer, diabetic retinopathy or exudative macular degeneration using the compounds of the invention.

Substituted phenols as active agents inhibiting VEGF production

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Skillman, NJ, United States of America

Soongyu Choi