A patient will stop receiving Translarna under the following ‘stop criteria’:

  • The Patient is non-compliant with assessments for continued therapy (non-compliance is defined as fewer than two attendances for assessment in any 14 month period).
  • If a patient has lost all ambulation (i.e. can no longer stand even with support) and has become entirely dependent on wheelchair use for all indoor and outdoor mobility (other than for reasons of an accident and/or an intercurrent illness), the patient’s physician needs to discuss stopping ataluren treatment.

In such cases as defined above, patients should stop treatment no later than 6 months after becoming fully non-ambulant. If you have any questions about the stop criteria, you should raise these with your physician.