A Call to Action

Patients with DMD have been specifically disadvantaged by the failure of the NHS England system to consider funding drugs for rare diseases. England was a leader in the trials for Translarna, in keeping with the government strategy to promote innovation and inward investment in trials. Just 80 patients might benefit from this drug if it is approved now, but every month a funding decision is delayed, boys lose eligibility for the drug to be prescribed.

Can the government show its commitment to patients with rare diseases by making an exceptional decision for the 80 boys for whom this treatment may otherwise not be available until too late? While guaranteeing that this situation will not arise again and that other rare disease indications will be speedily addressed in a streamlined and fair process in the future.

There are four things we urgently need your help with — please take action, sign petitions, write to your MPs, and make your voice heard in support of boys living with Duchenne who are running out of time.