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PTC Advances Cystic Fibrosis Candidate On Encouraging Data

This article was originally published in The Pink Sheet Daily

Executive Summary

Company is pursuing PTC124 in nonsense-mutation-mediated CF, a patient population of about 3,000 in the U.S.

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PTC Fully Enrolls Duchenne MD Trial; Expects Top-Line Data In Early 2010

With FDA approval of the six-minute walk test as primary endpoint, private specialty firm hopes to bring first Duchenne muscular dystrophy drug to market within two years.

PTC Fully Enrolls Duchenne MD Trial; Expects Top-Line Data In Early 2010

Despite targeting an indication with a very small population, PTC Therapeutics has fully enrolled a pivotal trial for ataluren (PTC124), its potential disease-modifying therapy for Duchenne muscular dystrophy, and expects to produce top-line data early next year. Regulatory filings in the U.S. and EU could follow closely if the data are promising

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