Ptc Therapeutics, Inc.

F. Hoffmann-la Roche Ag

Guangming Chen

Gary Mitchell Karp

Neil Gregory Almstead

Seongwoo Hwang

Young-Choon Moon

Richard Gerald Wilde

Nanjing Zhang

Anthony A Turpoff

James Jan Takasugi

Ellen Welch

Hongyu Ren

Xiaoyan Zhang

Chunshi Li

Jeffrey Allen Campbell

Jana Narasimhan

Peter Seongwoo Hwang

Steven David Paget

Matthew G Woll

Alexander Arefolov

Jin Zhu

Nikolai A Naryshkin

Hongyan Qi

Marla L Weetall

Anuradha Bhattacharyya

Xin Zhao

Amal Dakka

Tianle Yang

Song Xiao Huang

F George Njoroge

Emmanuel Pinard

Hasane Ratni

Suresh D Babu

Soongyu Choi

Aleksey I Gerasyuto

Nadiya Sydorenko

Chang-Sun Lee

Sergey Paushkin

Anthony R Mazzotti

Michael Andrew Arnold

Wuming Yan

Amin Nomeir

Sean Wesley Smith

Christie Morrill

Malcolm MacCoss

Malcolm Maccoss

Samit Hirawat

Langdon Miller

William Jospeh Lennox

Vara Prasad Venkata Nagendra Josyula

Jigar S Patel

Zhengxian Gu

Ronggang Liu

Ramesh Kakarla

Scott J Barraza

Jiashi Wang

Yalei Liu

Arthur Branstrom

Paul D Kennedy

Srinivasa Peddi

Olya Ginzburg

Md Rauful Alam

John David Baird

Concetta Freund

Anthony Turpoff

Yao Jiang

William Joseph Lennox

Christine Espiritu

Takashi Komatsu

Ellen M Welch

Maria L Weetall

Paul Kennedy

Jemes J Takasugi

Nikolai Naryshkin

Song Huang

Antony Allan Turpoff

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 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, 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.

Compounds for treating spinal muscular atrophy

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

The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.

1,3,4-oxadiazole benzoic acid compounds and their use for nonsense suppression and the treatment of disease

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

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

Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.

Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith

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

Pyrazole or triazole compounds and their use for the manufacture of a medicament for treating somatic mutation related diseases

Methods for using 1,2,4-Oxadiazole benzoic acid compounds

Growing community of inventors

Bridgewater, NJ, United States of America

Guangming Chen