#Open journalism No news is bad news

Your contributions will help us continue to deliver the stories that are important to you

Support The Journal
Dublin: 13°C Wednesday 23 September 2020

'He has a chance at life now': HSE grants access to drug that could help boys with rare condition keep walking

At least five boys in Ireland have Duchenne Muscular Dystrophy, a muscle-wasting condition.

Lewis Harte
Lewis Harte

A DRUG THAT could greatly improve the lives of young boys with a rare muscle-wasting condition has been approved for reimbursement by the HSE.

At least two boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) will benefit from access to Translarna. Their families have been fighting for access to the drug for years. 

Muscular Dystrophy Ireland (MDI) is aware of five boys in Ireland with the condition. Two of these boys are over five years of age and therefore eligible for Translarna, which is also known as Ataluren.

The remaining three boys may become eligible for the drug if they are still walking at the age of five. Most boys with the condition, which is more common in males, start to lose muscle power at the age of six, with the majority unable to walk by age 12.

The drug’s approval was confirmed in a letter sent by the HSE to MDI. It is already available in a number of other European countries. 

The letter, seen by TheJournal.ie, states: “The application for Translarna (Atlauren) was considered at the January 2019 Leadership Team meeting and was approved for reimbursement under a managed access arrangement as recommended by the Drugs Group.

“The company is being contacted to finalise the implementation arrangements for reimbursement.”

In 2016, the National Centre for Pharmacoeconomics (NCPE), which conducts the health technology assessment of pharmaceutical products for the HSE, did not to give the green light to Translarna

At the time it said the price would be over €400,000 per child per year, and didn’t deem it “cost-effective”. It’s understood a lower price tag has since been offered to the HSE following negotiations.

‘We’re walking on clouds’ 

Lewis Harte, from Castlebar in Co Mayo, is one of the boys who will benefit from the drug. TheJournal.ie has been following his story since 2016.

His mother Ann Marie Walsh-Harte found out the good news this evening. “We’re walking on clouds, I can’t believe that it has finally happened. We’re delighted,” she told us.  

Lewis Ann Marie and Lewis Source: MDI

Ann Marie she was afraid access to the drug would not be granted given ongoing issues in the health service, such as the spiralling cost of the National Children’s Hospital, saying: “Everything had just gone so quiet.”

She said Lewis, now seven years old, is doing well overall.  

Lewis is doing good, I’m so glad he’s still walking. You have to be walking in order to qualify for Translarna, we were just hoping that would still be the case if access was granted – and he is. 

“Mind-wise he is 100%, but his little body is letting him down, he hasn’t failed too much though and we’ve been able to keep him on his feet.”

Ann Marie said the family haven’t been given a timeline regarding access to the drug but she imagines it will happen “fairly rapidly” given the progressive nature of the condition. 

Time if not on our side, I imagine we’ll get it as soon as possible.

“It has been my whole life, trying to keep him walking and give him a good chance at life, I really hope he has that now. I hope other boys have that too.”

The other boy’s family have also been campaigning privately for access to Translarna but have remained anonymous. They are said to be “delighted” about the news.

Fianna Fáil TD James Browe is among those to have raised the issue in the Dáil. He told TheJournal.ie the HSE’s decision to grant access to the drug is “a huge relief to the families who have fought a long and hard battle for their children”.

About the author:

Órla Ryan

Read next:


This is YOUR comments community. Stay civil, stay constructive, stay on topic. Please familiarise yourself with our comments policy here before taking part.
write a comment

    Leave a commentcancel