Patient Registry Translarna (Ataluren)

This is a multicentre, observational study of patients receiving Translarna based on inclusion of their data in a registry. This study is intended to enrol approximately 200 patients across ~60 care centres in Europe and other regions over a period of ~ 2 years. The study population will include patients who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program) and who provide consent. Patients will be followed for at least 5 years from their date of enrolment. Safety and efficacy data will be collected in conjunction with routine visits conducted as per usual care. Although there are no protocol-mandated procedures, it is expected that physicians and other caregivers will follow published treatment guidelines and standards of care.

 
Primary Outcome Measures

• Incidence of adverse events.

 
Other Outcome Measures

• Changes in laboratory parameters.
• Changes in blood pressure.
• Prescriber and patient compliance with prescribing information according to the approved labelling.

 
Can I take part?

 
Inclusion Criteria

 
Exclusion Criteria