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PTC Therapeutics' Translarna shows positive effect in children with DMD

  • PTC Therapeutics (NASDAQ:PTCT) announces preliminary data from the first international drug registry for Duchenne muscular dystrophy (DMD) patients receiving Translarna (ataluren). The data were presented at the 23rd International Annual Congress of the World Muscle Society in Argentina.
  • The data show that children and adolescents receiving Translarna are continuing to walk years longer than untreated children, and are staying more physically able.
  • A time-to-event analysis for loss of ambulation has shown that patients on Translarna had a median age of loss of ambulation of 16.5 years of age – up to 5 years later than seen with natural disease progression in untreated children.
  • Patients who received Translarna in routine clinical practice also experienced a slower decline in their physical function compared with the placebo arm of Phase 3 Study 020, as measured by a series of timed function tests.
  • The analysis was based on data captured from 216 patients.

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