Adams urges access to Translarna for disabled boys with Duchenne Multiple Dystrophy
Sinn Féin President Gerry Adams TD today raised with the Taoiseach the availability of the drug Translarna for five young boys who are suffering from a rare form of Duchenne Multiple Dystrophy and who still fit the criteria for the drug. The Sinn Féin leader has also written to the HSE on this issue.
On Tuesday, Teachta Adams attended a briefing organised by Seanadóir Rose Conway Walsh and involving the parents of the young boys affected by this progressive, life limiting neuromuscular disease.
Speaking in the Dáil today Gerry Adams told the Taoiseach:
“The programme for Government is very clear that citizens with disabilities should be supported. Yesterday I attended a briefing in the hosted by Seanadóir Rose Conway-Walsh and attended by the parents of three wee boys, William, Cathal and Lewis. We were told the drug Translarna could make a real difference for these children. Five year old Lewis has been eligible for this medication for almost a year but he has not been able to access it as the Health Service Executive is refusing to reimburse the cost. Three year old Cathal, we are told, will lose the use of his legs and may need ventilation to help him breathe, and eventually his heart and lungs will fail. William faces a similar fate.”
The Sinn Féin leader asked the Taoiseach, in keeping with the Government's commitment to support citizens with disabilities, if he would instruct the Minister for Health and the HSE “to facilitate the availability of Translarna by agreeing to reimburse the cost and giving these lads a chance to fulfil the potential they have?”
In his response, the Taoiseach acknowledged that he has asked for more information on the decision although he also said that it was a matter for the HSE “to decide which drugs should be reimbursed and which are not”. ENDS
Note: In a submission by Dr Declan O’Rourke MD FRCPI Consultant Paediatric Neurologist in the Children’s University Hospital, Temple Street said:
“The findings of clinical trials show a favourable risk benefit profile for Translarna in patients with Duchenne muscular dystrophy, particularly when considering the serious and ultimately fatal nature of the disorder and the medical need for new Duchenne muscular dystrophy treatments. Slowing of a disease progression and motor decline is viewed by the Duchenne muscular dystrophy physician community as a realistic expectation of the effect of dystrophin restoration and patients and their care-givers consider this to be of highly valuable benefit of therapy. Treatment with Translarna may change the course of the disease and delay key milestones, potentially improving an overall life expectancy for patients.”