Ptc Therapeutics, Inc.

Westley J Friesen

Ellen Welch

Nikolai A Naryshkin

Charles Romfo

Meenal Patel

Yuki Tomizawa

Jin Zhuo

Neil Gregory Almstead

James Jan Takasugi

Richard Gerald Wilde

Paul D Kennedy

Jin Zhou

Ellen M Welch

The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular mystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).

Methods of screening for compounds for treating muscular dystrophy using human ALPHA7 integrin mRNA translation regulation

Methods of screening for compounds for treating muscular dystrophy using mIGF1 mRNA translation regulation

The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular dystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).

Methods of screening for compounds for treating muscular dystrophy using UTRN mRNA translation regulation

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

Growing community of inventors

Huntingdon Valley, PA, United States of America

Westley J Friesen