Does anyone know about BioMarin Utrophin Trials and why it was cancelled?  I thought this was very promising.   What happened?

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The results in healthy volunteers showed that there was no difference between those taking a placebo and those taking the drug in terms of utrophin upregulation

Discovery of 2-Arylbenzoxazoles as Upregulators of Utrophin Production for the Treatment of Duchenne Muscular Dystrophy

New candidate to utrophin upregulation to cure Duchenne:

A series of novel 2-arylbenzoxazoles that upregulate the production of utrophin in murine H2K cells, as assessed using a luciferase reporter linked assay, have been identified. This compound class appears to hold considerable promise as a potential treatment for Duchenne muscular dystrophy. Following the delineation of structureactivity relationships in the series, a number of potent upregulators were identified, and preliminary ADME evaluation is described. These studies have resulted in the identification of 1, a compound that has been progressed to clinical trials

The publication you refer to summarises all the years of chemistry effort from the first utrophin up regulation screen performed by Summit to the final compound, SMT C1100, which Summit are looking to reformulate and re-enter into Phase I clinical trials. Jon
Jon, you mentioned that PPMD is evaluating your proposal. What about other orgs? When do  you expect to hear anything from MDA, or European organizations where Summit might have applied for funds as well?

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