PTC Therapeutics recently presented positive preliminary data on the long-term clinical benefit of Translarna at the World Muscle Society conference. Data collected from 216 treated children showed that some boys were able to walk up to five years longer compared to untreated children (based on natural history data).
PTC Therapeutics has been working in partnership with TREAT-NMD to collect this information through the STRIDE registry. No long-term safety concerns of Translarna were reported.
For further information pleases read PTC Therapeutics’ press release.