Ptc Therapeutics, Inc.

F. Hoffmann-La Roche Ag

Fmc Corporation

Nanjing Zhang

Gary Mitchell Karp

Xiaoyan Zhang

Jana Narasimhan

Anthony A Turpoff

Guangming Chen

Matthew G Woll

Nikolai A Naryshkin

Hongyan Qi

Amal Dakka

Tianle Yang

Chunshi Li

Emmanuel Pinard

Hasane Ratni

Ellen Welch

Soongyu Choi

Marla L Weetall

Xin Zhao

Steven David Paget

Hongyu Ren

Michael Andrew Arnold

Luke Green

James Jan Takasugi

Richard Gerald Wilde

Anuradha Bhattacharyya

Jiashi Wang

Jin Zhu

Young-Choon Moon

Jeffrey Allen Campbell

Suresh D Babu

Aleksey I Gerasyuto

Peter Seongwoo Hwang

Nadiya Sydorenko

Alexander Arefolov

Anthony R Mazzotti

Sean Wesley Smith

Song Xiao Huang

F George Njoroge

Chang-Sun Lee

Vara Prasad Venkata Nagendra Josyula

Sergey Paushkin

Andrew Kassick

Jigar S Patel

Wuming Yan

Amin Nomeir

Scott J Barraza

Christie Morrill

Md Rauful Alam

Arthur Branstrom

John David Baird

Olya Ginzburg

Srinivasa Peddi

Nikolai Naryshkin

Malcolm MacCoss

George Theodoridis

Harvey Wendt

Zhenrong Xu

Malcolm Maccoss

William Jospeh Lennox

John Francis Chiarello

Ramil Y Baiazitov

Wu Du

Ramesh Kakarla

Ronggang Liu

Lukiana Amedzo

Yalei Liu

Tamil Arasu

Anthony A Yurpoff

Nathaniel T Kenton

Sudipta Pal

Nicholas W Mszar

Tom Tuan Luong

Anthony Turpoff

Yi Jin Kim Gorske

William Joseph Lennox

Yao Jiang

Nicholas Walter Mszar

Rauful Alam

Maria L Weetall

Song Huang

Gang Wang

Antony Allan Turpoff

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 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.

Compounds for treating 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.

Provided herein are compounds of Formula (I): 

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.

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

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.

Compounds and methods for antiviral treatment

Growing community of inventors

Princeton, NJ, United States of America

Nanjing Zhang