For the past year The Trust has been engaging with key stakeholders in relation ensuring that once treatments become available for children and adults with Duchenne they can be accessed immediately. Unfortunately at present this process is complex and frustratingly slow. We are in the process of organising more meetings in this regard and we shall keep you informed of our progress.
At present we are supporting the campaign to ensure that the drug Translarna is made available to those eligible in the Republic of Ireland. We are advised that at present two children need this drug. Translarna is an exon skipping drug that has the potential to slow the disease down for people who have something called a nonsense mutation. It is essential that this drug and future drugs are accessible to those who could benefit.
It is tragic enough having a child with a terminal disease without then having to engage in petitions and seeking help through the media. This will not cost you anything but a minute of your day. I know the families involved would be so grateful to you.
Please help support these children by signing this petition: