The Government has been urged to approve a new treatment for patients with Duchenne muscular dystrophy.
The support group Muscular Dystrophy Ireland has also called on the Government to radically improve access to clinical trials for new and emerging therapies.
It said there was no centre for research or clinical trials in Ireland and patients with the condition had to go to the UK to access the latest therapies.
Duchenne muscular dystrophy is a serious muscle-wasting condition that primarily affects boys.
Muscular Dystrophy Ireland said the new drug treatment, known as "Translarna", had received conditional approval from the European Medicines Agency for specific Duchenne patients whose condition was caused by a particular genetic defect in the dystrophin gene.
Keeping boys on their feet
“It is the first treatment to address the underlying genetic cause of Duchenne muscular dystrophy, slowing its progression in patients aged five years and older who are able to walk. It is available in a number of countries in the
European Union
but has yet to be offered in Ireland.”
The group said Duchenne muscular dystrophy affected one in 3,500 male births.
“Typically boys with Duchenne lose their ability to walk between the ages of 10 and 14 years; by their late teens, they will lose their strength in their upper bodies, including movement in their arms, while the progressive muscle weakness also leads to serious medical problems, particularly with the heart and lungs.”
For the first time this year medical clinics are to be provided at Beaumont Hospital in Dublin for young adults with the condition.
‘The forgotten condition’
Muscular Dystrophy Ireland chief executive
Joe Mooney
said: “For too long, Duchenne muscular dystrophy has been the forgotten condition.
“The most common and the most severe type of muscular dystrophy, it is a condition so insidious that it creeps up on boys who are initially able to walk and do all the fun things that other boys do, then slowly but relentlessly takes their mobility away as they enter their teenage years.
“It leaves them confined to a wheelchair in their teens when other young people are experiencing all that life has to offer.
“We now have the first new therapy, Translarna, that has the potential to keep some young boys with Duchenne on their feet for longer. It would also make them eligible for other subsequent treatments that require them to be mobile. There shouldn’t need to be a discussion about this. We need the Government to take this issue seriously and to approve its use for the small number of people who need it.”
