Translarna first ever drug will treat 50 boys in England
We were thrilled to hear the news last Friday, that NICE have given the green light on Translarna – the first ever drug to treat the underlying cause of Duchenne for those eligible. Whilst this is great news for those it will help 50 boys in England, it will not help all. We have 2,500 […]
Super media coverage
We would like to thank Rachel Hargraves from RDZ-PR who is donating her time to Alex’s Wish – a true Wish Warrior – to raise awareness of our cause. Through this relationship we have generated a great deal of media coverage in the last week across the Leicestershire region in both the Leicester Mercury and Loughborough Echo. We now […]
Vigil for Translarna
On Wednesday, 11th February, Alex’s Wish took part in a vigil at the door of the NHS England alongside a number of Duchenne charities, families and supporters to demand access to a new ground-breaking drug Translarna. Translarna is the first drug to be approved that offers the prospect of delaying disease progression and keeping boys […]
Urgent Call to Action – Translarna
Patients with Duchenne Muscular Dystrophy 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 […]
Translarna Lobby
Alex’s Wish took part in a lobby at Westminster on Wednesday, 14th January with the aim to ensure early availability and full funding of a pioneering new treatment called Translarna. Translarna is the first drug to address the underlying cause of Duchenne. We were welcomed by Dave Anderson MP, Chair of the All Party Parlimentary […]