Ptc Therapeutics, Inc.

American Cyanamid Company

F. Hoffmann-la Roche Ag

Other

Hoffmann-la Roche Inc.

Gary Mitchell Karp

Guangming Chen

Neil Gregory Almstead

Young-Choon Moon

Nanjing Zhang

Jana Narasimhan

Seongwoo Hwang

Anthony A Turpoff

Xiaoyan Zhang

Matthew G Woll

Ellen Welch

Nikolai A Naryshkin

Hongyan Qi

Richard Gerald Wilde

Philip Mark Harrington

Hongyu Ren

Amal Dakka

Michael A Guaciaro

Tianle Yang

Hasane Ratni

Marla L Weetall

Mark C Manfredi

Chunshi Li

Emmanuel Pinard

Michael E Condon

Jeffrey Allen Campbell

Soongyu Choi

James Jan Takasugi

Xin Zhao

Peter Seongwoo Hwang

Anuradha Bhattacharyya

Luke Green

Suresh D Babu

Steven David Paget

Alvin D Crews, Jr

Alvin Donald Crews

Alexander Arefolov

Michael Andrew Arnold

Anthony R Mazzotti

Jigar S Patel

Simon David Gill

Jiashi Wang

Petra Dieterich

Scott J Barraza

Jin Zhu

Zhenrong Xu

Aleksey I Gerasyuto

Nadiya Sydorenko

Chang-Sun Lee

Sean Wesley Smith

Lukiana Amedzo

Song Xiao Huang

F George Njoroge

Vara Prasad Venkata Nagendra Josyula

Sergey Paushkin

Axel Kleeman

Wuming Yan

Alvin Donald Crews, Jr

Amin Nomeir

Jeffrey H Birk

Christie Morrill

Arthur Branstrom

John David Baird

Olya Ginzburg

Md Rauful Alam

Srinivasa Peddi

Andrew J Kassick

Maria L Weetall

Malcolm MacCoss

Thomas Maier

Stefan Scheiblich

Malcolm Maccoss

Langdon Miller

Samit Hirawat

William Jospeh Lennox

Axel Kleemann

Ramil Y Baiazitov

Wu Du

Zhengxian Gu

Ramesh Kakarla

Ronggang Liu

Yalei Liu

Paul D Kennedy

Hiroo Koyama

Tamil Arasu

Concetta Freund

Takashi Komatsu

Nicole Risher

William Joseph Lennox

Yao Jiang

Halmut Siegfried Baltruschat

Anthony A Yurpoff

Yi Jin Kim Gorske

Nathaniel T Kenton

Sudipta Pal

Nicholas W Mszar

Tom Tuan Luong

Anthony Turpoff

Christine Espiritu

Nicholas Walter Mszar

The present description relates to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect described herein relates to substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.

Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

Compounds for treating Huntington's disease

The present description relates' to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.

Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted benzothiazole compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

Heteroaryl compounds for treating Huntington's disease

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

Heterocyclic and heteroaryl compounds for treating Huntington's disease

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I) or Formula (II), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

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

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heteroaryl compounds of Formula (I), Formula (II), Formula (III), or Formula (IV), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. 

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.

Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.

Compositions of 1,2,4-oxadiazole benzoic acid compounds and methods for their use

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.

Growing community of inventors

Princeton Junction, NJ, United States of America

Gary Mitchell Karp