A new treatment for people with a particular type of Duchenne muscular dystrophy has not been recommended for reimbursement in Ireland.

There are around 30 types of the neuromuscular condition, muscular dystrophy. Duchenne is the most common and most severe type, mainly affecting boys. It affects around one in every 3,500 male births and usually boys with the condition lose their ability to walk between the ages of 10 and 14. By their late teens, they tend to lose strength in their upper bodies, including their arms.

The progressive muscle weakness associated with this condition also leads to other serious health issues, such as heart and lung problems.

Translarna is a new treatment for people with a certain type of Duchenne muscular dystrophy. Specifically, it is aimed at those whose condition is caused by a particular genetic defect - known as a nonsense mutation in the dystrophin gene - and who are aged five and older and still able to walk.

According to Muscular Dystrophy Ireland (MDI), Translarna is the first treatment to address the underlying genetic cause of Duchenne muscular dystrophy.

It has already been approved for use in England for a period of time to allow further evidence to be collected on its use. It is also the first Duchenne-targeting treatment to receive conditional marketing approval by the European Medicines Agency (EMA).

It is currently available and being prescribed to boys in more than 20 EU countries. Some have been receiving it since late 2014.

However, the National Centre for Pharmacoeconomics (NCPE), which facilitates healthcare decisions on the reimbursement of treatments using clinical and scientific evidence, has decided not to recommend Translarna for reimbursement here.

According to John Bennett of MDI, ‘it is only fair and reasonable' that Irish children should have the same opportunities as children elsewhere in Europe.

"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. 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.

"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," he insisted.

While this recommendation has been made by the NCPE, it is the HSE that will make the final decision on reimbursement. MDI is calling on the HSE to enter into discussions with the manufacturer and come to some sort of arrangement that will make Translarna available in Ireland.

For more information on muscular dystrophy and MDI, click here