Families have been waiting a long time for new treatments to come on stream and everyone in the Duchenne community urges the NHS in England, Scotland, Wales and Northern Ireland to fully fund the costs of Translarna and support its immediate delivery to patients.
Why is this important?
Translarna is the first novel drug to be granted market approval for Duchenne Muscular Dystrophy (DMD) that treats the underlying causes of the condition. DMD is a life limiting condition for which there is no cure. The severe and progressive muscle wasting leaves young adults reliant on wheelchairs, ventilators and at severe risk of early heart failure. The US company PTC Therapeutics have been granted marketing authorisation in the European Union under the trade name Translarna for the treatment of Duchenne for a sub group of patients aged five years and older who are walking and have a single point variation in their dystrophin gene.
This is a crucial landmark in treating Duchenne. Translarna is expected to be the first of other personalised medicines that are currently in clinical trial and could also become available to treat more young people with DMD.
It is now imperative that all children who are identified with these specific Duchenne gene mutations have long term access to this novel therapy.
