‘It can give children with DMD a longer life expectancy’ mother

Parents from South Dublin attended a peaceful demonstration on Wednesday asking the Government to speed up their approval of a life-changing medicine.

One month ago, the European Medicines Agency granted marketing authorisation to Givinostat, which is already used in the UK and the US to delay progression of Duchenne Muscular Dystrophy (DMD).

Tallaght woman Una Ennis, mother of Archie (7) has attended the protest as Archie’s battle with DMD since last December has touched everyone in the community, and she has finally found hope.

“This is to put pressure on our Government to get the medicine quickly from the pharmaceutical companies,” she said ahead of the demonstration.

“The data have shown what it can do. It can give children with DMD a longer life expectancy and keep them on their feet for longer.

“While there’s treatment available for children with other conditions, children with Duchenne are just handed a wheelchair and then left on their own.”

Parents like Una are concerned about the lengths of Irish bureaucracy which could take too long to turn the EMA’s approval into an actual delivery of the medicine in the country.

According to a message Una released online, approval from the National Centre for Pharmacoeconomics (NCPE) is needed before the Government can get the drug from the pharmaceutical companies that started producing it for EU distribution.

“Every day that Archie doesn’t have any medicine, his muscles are dying,” continued Una.

“This would get my boys and everyone around them a better quality of life,” said another mother from Lucan, Karen Thompson, whose both boys Conor (9) and Dean (8) are living with DMD.

The family recently had to move to a new, adaptable home as the one they were living in had become unsuitable for the boys’ needs.

“This is the first hope any family had in years,” said Karen. “I was at the demonstration to highlight the huge need for something that is already available in other countries while Ireland is falling behind.

“We don’t have time to wait for the usual bureaucracy. Every single day matters in Duchenne. The Government need to understand that these children are losing abilities every day that passes. They can’t get their muscles back.”

Families feel they’re running out of time because the EMA’s approval was for treatment “in patients from the age of six who are able to walk.”

“My youngest is still okay, but my eldest is already using a wheelchair at school and for longer distances,” continued Karen.

“And we know that as children with this condition are prone to falling, they could break a bone and stop walking any day.

“We are working against the clock.”

DMD is a rare degenerative condition that causes rapid, progressive muscle degeneration and weakness, starting from the legs, hips and shoulders, until it reaches the lungs causing respiratory difficulties and finally the heart.

Givinostat, the active substance of a medicine called Duvyzat, can inhibit one of the enzymes responsible for the condition and this may help reduce inflammation and tissue scarring and thickening.