Ptc Therapeutics, Inc.

Paul D Kennedy

Neil Gregory Almstead

Ellen Welch

James Jan Takasugi

Richard Gerald Wilde

Gary Mitchell Karp

Young-Choon Moon

Guangming Chen

Peter Seongwoo Hwang

Samit Hirawat

Langdon Miller

Hongyu Ren

Westley J Friesen

Ellen M Welch

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

The invention encompasses nucleoside compounds, compositions comprising the compounds and methods for treating or preventing diseases associated with nonsense mutations of mRNA by administering these compounds or compositions.

Nucleoside compounds and their use for treating cancer and diseases associated with somatic mutations

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Kalamazoo, MI, United States of America

Paul D Kennedy