Ptc Therapeutics, Inc.

F. Hoffmann-La Roche Ag

Fmc Corporation

Hoffmann-La Roche Inc.

Bayer Cropscience Ag

Pct Therapeutics, Inc.

Hongyan Qi

Soongyu Choi

Gary Mitchell Karp

Young-Choon Moon

Jana Narasimhan

Tianle Yang

Xiaoyan Zhang

Nanjing Zhang

Marla L Weetall

William Jospeh Lennox

Matthew G Woll

Emmanuel Pinard

Hasane Ratni

Nikolai A Naryshkin

Amal Dakka

Ellen Welch

Liangxian Cao

Anthony A Turpoff

Xin Zhao

Donald Thomas Corson

Luke Green

Nadarajan Tamilarasu

Seongwoo Hwang

George Theodoridis

Guangming Chen

Thomas W Davis

Nikolai Naryshkin

Walter H Yeager

Neil Gregory Almstead

Thomas G Cullen

Aleksey I Gerasyuto

Peter Seongwoo Hwang

Daniel H Cohen

Vara Prasad Venkata Nagendra Josyula

Edward J Barron

Chang-Sun Lee

Syed F Ali

Chunshi Li

Sergey Paushkin

Michael Andrew Arnold

Elizabeth Gerig Rowley

Jiashi Wang

Sean Wesley Smith

Song Xiao Huang

Srinivasa Peddi

Olya Ginzburg

John David Baird

Tamil Arasu

Ellen M Crawford

Christina B Duggan

Arthur Branstrom

Duck-Hyung Lee

William Lennox

Jeff Campbell

Maria L Weetall

Jeffrey Allen Campbell

Richard Gerald Wilde

David M Roush

Langdon Miller

Christopher Robert Trotta

John Francis Chiarello

Wu Du

Zhengxian Gu

Harry H Miao

Matthew Woll

Jason D Graci

Anthony A Yurpoff

Nicole Risher

Don Corson

Arasu Tamil

Yi Jin Kim Gorske

Christopher Trotta

Neil Almstead

Song Huang

Gary Karp

William Joseph Lennox

Maria Weetall

Thomas Davis

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 improved processes for the synthesis of substituted tetrahydro beta-carboline derivatives. In particular, provided herein are improved processes useful for the preparation of (S)-4-chlorophenyl 6-chloro-1-(4-methoxyphenyl)-3,4-dihydro-1H-pyrido[3,4-b]indole-2(9H)-carboxylate. Formula (I): 

Processes for the preparation of substituted tetrahydro beta-carbolines

Provided herein are compounds of Formula (I): 

The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments. 

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, compositions thereof and uses therewith for treating spinal muscular atrophy.

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

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.

Antibacterial compounds and methods for use

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

Growing community of inventors

Plainsboro, NJ, United States of America

Hongyan Qi