Nic Bungay, Muscular Dystrophy’s UK’s Director of Campaigns, Care and Information, said:
“This is disappointing news for the people with Duchenne muscular dystrophy who could benefit from Raxone. With no alternative treatment to help preserve respiratory function when steroids stop working, adults with Duchenne can struggle to cough and clear their airways.
“Raxone offered many people with Duchenne hope when it was made available via the Early Access to Medicines Scheme. We are in close contact with Santhera about what happens next for those patients in the UK currently receiving Raxone, as part of the EAM scheme. We will today write to the MHRA urging them to ensure that patients who are benefiting from the drug do not have it withdrawn.
“Duchenne has few treatment options and no cure. We must continue to look at innovative ways of bringing promising Duchenne treatments at the earliest possible stage to those who could benefit from them.”