Please make Translarna available in Ireland to help boys with Duchenne Muscular Dystrophy
0 have signed. Let’s get to 15,000!
This petition is on behalf of parents of children suffering with Duchenne Muscular Dystrophy (DMD). Our three year old son has this rare condition and we would love to see him (and other boys suffering with DMD) having access to a potentially helpful treatment called Translarna.
Translarna is the first novel drug to be granted market approval for 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.
Please sign this petition to make this potentially very helpful treatment available in Ireland.
Today: Nick is counting on you
Nick Jones needs your help with “Simon Harris: Please make Translarna available in Ireland to help boys with Duchenne Muscular Dystrophy”. Join Nick and 13,708 supporters today.