Government Called On To Facilitate Access To Treatment For Children With Muscular Dystrophy

On World Duchenne Awareness Day, Muscular Dystrophy Ireland is expressing disappointment, at the HSE decision not to fund a life changing drug for children with Duchenne Muscular Dystrophy. This news follows a lengthy assessment process that has spanned almost two years.

Richard Lodge, Chief Executive Officer of Muscular Dystrophy Ireland comments

“Translarna is specifically for those children whose condition is caused by a particular genetic defect, called a nonsense mutation in the dystrophin gene, and who are aged five years and older and still able to walk.

“A limited number of patients will benefit from this drug, if reimbursed, so while the

individual cost of the drug is high, the budget impact is relatively low”

“It has been disappointing and frustrating for the families involved. Some of whom are seriously considering emigration, to get the best possible care for their sons”

Translarna has been reimbursed in the UK for over a year now, including Scotland, Northern Ireland and the Isle of Mann.

“That is why today we are calling on the Minister for Health, Simon Harris TD, to intervene and to ensure that Irish boys that desperately need this therapy receive it without further delay and have the same opportunities as other children across Europe.”

Last in Europe

“Since 2014, over 400 children in 22 European countries have been receiving this treatment. That means that 80% of eligible children are now receiving the drug and this leaves Ireland as one of the last remaining countries to facilitate treatment.”

“It would appear that Ireland’s decision not to reimburse for Translarna is at odds with regulators, health authorities and experts across Europe. Would this imply that the European Medicines Agency and the 22 other European Countries have all gotten it wrong? Or is it just that we are behind the curve?”

Anne Marie Harte is the mother of five year-old Lewis, from Castlebar in County Mayo.   For Lewis, Translarna is the only treatment option.
“It is so unfair knowing that there is a treatment out there that can help our son but it is being denied to him. This is a race against time that we don’t have. It’s simply agonising.”

For more information on muscular dystrophy and the work of Muscular Dystrophy Ireland, visit www.mdi.ie or follow on Facebook at facebook.com/mdiireland and on Twitter @MDI_ireland.

About Muscular Dystrophy Ireland

Muscular Dystrophy Ireland (MDI) is a national organisation which was established in 1972 by a small group of people in the west of Ireland to support families who had a member with muscular dystrophy. Since then it has grown considerably and it now has a network of branches throughout Ireland.

MDI provides expert information, support and services to people with muscular dystrophy and their families. We advocate for services to enable people to participate in society and to live independently. We offer extensive information and guidance to education providers and stakeholders within local communities who wish to support inclusion of people with muscular dystrophy. We partner closely with leading medical professionals to provide a holistic support service to our members. We facilitate and support research into muscular dystrophy to develop knowledge of the condition and to further the collective effort towards better care and treatment.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is one of more than 30 types of muscular dystrophy and affects approximately one in 3,500 male births. It arises due to the absence of dystrophin, a gene found on the X chromosome, vital in holding muscles together and results in a progressive loss of muscle strength, leading to paralysis, heart and lung failure.

As boys have only one X chromosome, they are more susceptible to dystrophin damage, unlike girls who have a second X chromosome to make up for the damaged gene. For a woman who carries the Duchenne muscular dystrophy gene, there is a one-in-two chance that her sons will have the condition and a one-in-two chance that her daughters will carry the gene. Approximately one-in-three cases arise due to a random genetic mutation meaning that it can affect anyone.

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. As research efforts continue around the world, and new treatments can significantly slow the progression of the condition, regrettably, there is no cure.