Muscular Dystrophy Ireland disappointed with NCPE decision

“It is only fair and reasonable to expect Irish children to have the same opportunities as the children in other european countries who have access to this medication,” said Mr John Bennett of MDI.

“It is our information that this new treatment would only apply in Ireland to a very small number of patients in the low single digits.”

Translarna is a new treatment for people with a particular type of Duchenne Muscular Dystrophy. MDI has raised access to this therapy on a number of occasions in recent months, including on World Duchenne Awareness Day, and in various meetings with public representatives and other stakeholders.

“However, for those young people it would make a huge difference in slowing progression of the condition and in keeping them on their feet for longer,” said Mr John Bennett of MDI.

“It means that they may experience more of what life has to offer and delay the requirement for other medical and social care supports. For young people and their families whose quality of life and life expectancy is significantly impacted by this condition, this is of immense value.”

MDI noted that while this recommendation has been made by the NCPE, the final decision on reimbursement rests with the HSE.

“We are now calling on the HSE to enter into discussions with the manufacturer and to come to an arrangement which would make the therapy available here and to do so in as timely a manner as possible,” MDI Ireland said in their statement today.