The treatment ataluren (Translarna) for Duchenne muscular dystrophy has now exited managed access and entered the NICE re-evaluation process. In order to provide some reassurance to those already receiving ataluren through managed access, NICE and NHSE have worked to produce the below statement:
NHS England and PTC therapeutics have agreed an arrangement to ensure continuity of care for patients who are currently treated with ataluren as part of the managed access agreement, after the managed access agreement has expired, where clinically appropriate. Therefore, patients who are currently treated with ataluren in the managed access agreement will continue to be able to access treatment, if this is deemed clinically appropriate, should NICE be unable to make a positive NICE recommendation.
Please contact us info@actionduchenne.org if you have any questions.
Insights from a Duchenne Grandad
