Ptc Therapeutics, Inc.

F. Hoffmann-la Roche Ag

Hoffmann-la Roche Inc.

Ptc Theranentics. Inc.

Pct Therapeutics, Inc.

Marla L Weetall

Nikolai A Naryshkin

Hasane Ratni

Ellen Welch

Gary Mitchell Karp

Luke Green

Hongyan Qi

Jana Narasimhan

Amal Dakka

Emmanuel Pinard

Soongyu Choi

Xin Zhao

Matthew G Woll

Xiaoyan Zhang

Liangxian Cao

Nanjing Zhang

Tianle Yang

Anthony A Turpoff

Thomas W Davis

Langdon Miller

Guangming Chen

James Jan Takasugi

Samit Hirawat

Mandar V Dali

Chunshi Li

Harry H Miao

Chang-Sun Lee

Sergey Paushkin

Song Xiao Huang

Young-Choon Moon

Neil Gregory Almstead

Jeffrey Allen Campbell

Peter Seongwoo Hwang

William Jospeh Lennox

Christopher Robert Trotta

Charles Romfo

Donald Thomas Corson

Zhengxian Gu

Tamil Arasu

Jason D Graci

Anthony A Yurpoff

Kenneth P Olive

Melissa L Dumble

Jaime A Eberle-Singh

Nicole Risher

Thomas Davis

Don Corson

Jeff Campbell

William Lennox

Song Huang

A method for treating pancreatic cancer in a subject in need thereof comprising, administering to the subject an effective amount of a tubulin polymerization inhibitor compound is described herein. More particularly, a method for treating pancreatic ductal adenocarcinoma in a subject in need thereof comprising, administering to the subject an effective amount of a substituted reverse pyrimidine tubulin polymerization inhibitor compound alone or in combination with other chemo-therapeutic agents is described herein.

Method for treating pancreatic cancer

The present invention provides compounds of formula (I)

Compounds for treating spinal muscular atrophy

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.

A method of use for Cpd 1 as an inhibitor of dihydroorotate dehydrogenase (DHODH) function in treating or ameliorating a hematological cancer in a subject in need thereof comprising, administering an effective amount of Cpd 1 to the subject, having the structure: or a form or pharmaceutical composition thereof.

DHODH inhibitor for use in treating hematologic cancers

The present invention provides compounds of formula (I) 

Provided herein are compounds of Formula (I): 

Provided herein are pharmaceutical compositions, which comprise a 1,2,4-oxadiazole benzoic acid or a pharmaceutically acceptable salt thereof. Further provided herein are certain pharmaceutically acceptable salts of a 1,2,4-oxadiazole benzoic acid and methods for making the same. Further provided herein are methods of treating or preventing a disease associated with a nonsense mutation or a premature stop codon, comprising administering such pharmaceutical compositions or pharmaceutically acceptable salts to a patient having a disease associated with a nonsense mutation or a premature stop codon.

Pharmaceutical compositions and salts of a 1,2,4-oxadiazole benzoic acid

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

Growing community of inventors

Morristown, NJ, United States of America

Marla L Weetall