Ptc Therapeutics, Inc.

F. Hoffmann-la Roche Ag

Anthony A Turpoff

Gary Mitchell Karp

Nanjing Zhang

Guangming Chen

Jana Narasimhan

Xiaoyan Zhang

Matthew G Woll

James Jan Takasugi

Hongyan Qi

Richard Gerald Wilde

Hongyu Ren

Tianle Yang

Chunshi Li

Emmanuel Pinard

Hasane Ratni

Jeffrey Allen Campbell

Ellen Welch

Nikolai A Naryshkin

Soongyu Choi

Marla L Weetall

Peter Seongwoo Hwang

Anuradha Bhattacharyya

Xin Zhao

Amal Dakka

Alexander Arefolov

Suresh D Babu

Steven David Paget

Anthony R Mazzotti

Jin Zhu

Young-Choon Moon

Luke Green

Nadiya Sydorenko

Jigar S Patel

Scott J Barraza

Song Xiao Huang

Zhenrong Xu

Aleksey I Gerasyuto

Chang-Sun Lee

Sergey Paushkin

Michael Andrew Arnold

Wuming Yan

Sean Wesley Smith

Lukiana Amedzo

Andrew J Kassick

Md Rauful Alam

F George Njoroge

Malcolm MacCoss

Vara Prasad Venkata Nagendra Josyula

Ramil Y Baiazitov

Wu Du

Zhengxian Gu

Ronggang Liu

Ramesh Kakarla

Jiashi Wang

Amin Nomeir

Yalei Liu

Christie Morrill

Srinivasa Peddi

Olya Ginzburg

John David Baird

Tamil Arasu

Arthur Branstrom

Nicholas W Mszar

Sudipta Pal

Nathaniel T Kenton

Yi Jin Kim Gorske

Yao Jiang

William Joseph Lennox

Takashi Komatsu

Concetta Freund

Christine Espiritu

Song Huang

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

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): 

Compounds for treating spinal muscular atrophy

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.

Indole derivatives and methods for antiviral treatment

In accordance with the present invention, compounds that inhibit viral replication, preferably Hepatitis C Virus (HCV) replication, have been identified, and methods for their use provided. In one aspect of the invention, compounds useful in the treatment or prevention of a viral infection are provided. In another aspect of the invention, compounds useful in the treatment or prevention of HCV infection are provided.

Methods for treating hepatitis C

The present invention provides compounds, pharmaceutical compositions, and methods of using such compounds or compositions for treating infection by a virus, or for affecting viral IRES activity.

Growing community of inventors

Hillsborough, NJ, United States of America

Anthony A Turpoff