PARENTS of children suffering from a rare muscular disease are pleading with the government to speed up the approval of a new miracle drug that could help them live longer and retain the ability to walk.
Duchenne Muscular Dystrophy is a rare condition that mainly affects boys and causes progressive muscle loss as they grow older.
The life-limiting disease – which effects fewer than 200 children in Ireland – results in kids losing the ability to walk and use their muscles as it progresses.
There is no cure for the disease but a new drug has been invented that significantly delays the progression of the disease and gives children more time.
Eilish and Mark O’Sullivan, from Co Donegal, have two boys with Duchenne Muscular Dystrophy.
The family were told that their oldest son Nathan would lose the ability to walk by 12 and may not live past the age of 18.
However, the GAA-mad youngster was admitted to a clinical trial in the UK for the miracle drug Givinostat and is now 21 years old and still able to walk.
Nathan’s younger brother Diarmuid is nine years old and is not able to access the same drug because it has not yet been approved in Ireland – despite already being rolled out in other countries across the world.
Mum Eilish and dad Mark joined around 100 people at a protest outside Leinster House today where families are pleading with Health Minister Jennifer Carroll McNeill to step in and speed up the approval of this drug in Ireland.
Eilish told the Irish Sun: “When Nathan was diagnosed, we brought him over to Newcastle in the UK for a clinical trial back in 2016.
“He’s been on the drug ever since. He was previously told he wouldn’t be able to walk by 12 and he could possibly lose his life by his 18th birthday.
“He is now 21 and he does the score board for all the Donegal GAA matches in MacCumhaill Park in Ballybofey and climbs up and down the steps to do it. It is a miracle and we put that down to the drug.”
MIRACLE MEDICINE
The loving parents brought a dose of Nathan’s miracle medicine to the protest today to highlight the heartbreaking situation where one of their sons can access this drug while his younger brother can’t.
Dad Mark said: “We’ve seen what this drug can do in one boy and yet in the same home the other boy doesn’t have the same opportunity.
“That is hard to explain to him because when the time comes and he asks ‘how come my brother is 21 or 22 and I’m nine, ten or 11 and I can’t walk but he seems to be doing OK.”
The roll out of the drug in Ireland is incredibly time sensitive for children with muscular dystrophy as it has only been approved for use by the European Commission for children who are still able to walk.
Several parents at the protest today told the Irish Sun that they fear the HSE will take so long to approve the medicine that their sons will no longer be able to walk and will no longer be eligible for the treatment.
‘DRUG SLOWS PROGRESSION’
Little Dean Thompson, eight, and his brother Conor, 10, both live with Duchenne Muscular Dystrophy.
Conor has started using a power wheelchair as the muscles on his legs deteriorate while his younger brother is still without the need for a chair.
Dad Jamie told the Irish Sun: “This drug slows down the progression of Duchenne. We know it’s not a cure.
“But every day our boys go without this drug, our boys lose a little bit more muscle and they will not get that back.
“This will change lives. It is the difference of the lads staying on their feet for another few years and that is huge.”
‘FULL OF LOVE FOR LIFE’
Mum Karen added: “Conor and Dean are amazing little boys. They are full of love for life. They make us laugh every day.
“We want them to live their best life for as long as they can. They enjoy life. They have great pals in school and friends.
“They love swimming and playing with their brothers and gaming like all kids. They are so caring, thoughtful and loving and we just want to hold onto them as long as we can.
“We really really need this drug. They need to approve it as soon as possible because our boys don’t have time to wait. Every day is a day lost.
‘THEY NEED IT NOW’
“They are using this in other countries already all over Europe. There are early access programmes. There are compassionate access programmes.
“It is not fair to make our boys wait. They need it now.
“We don’t have time. If Conor has to wait another year, the chances are he won’t be eligible for this.”
“We’ve seen what this drug can do in one boy and yet in the same home the other boy doesn’t have the same opportunity.”
Mark O’Sullivan
Little Fionn Brogan is nine years old from Dublin and was diagnosed with Duchenne six years ago.
Mum Maeve told the Irish Sun: “Fionn is incredibly stubborn and determined. He’s really behind the campaign because he wants people to see how strong boys with Duchenne are.
“We call them our warriors because they have to keep fighting.
“Fionn is at the stage where he is really struggling with things and finding steps and stairs hard.
“He is on the edge. If this drug takes too long to get through then he could miss out on it.”
‘WE CAN’T WAIT’
Dad Michael is pleading with the Health Minister to step in and set up a pilot programme immediately to enable children like Fionn to get access to this drug in the coming months.
He said: “We want a very concrete plan. I want to see the commissioning of a pilot programme to expedite the rapid access of the drug.
“It is likely that it could take up to two years to approve in our system here. We can’t wait that long.
“There’s no reason why the Minister can’t intervene here immediately and introduce a pilot programme to roll out the drug now.
“I’ve spoken to the clinicians who would be involved in rolling out such a programme and they said they are willing and able to do it.”
URGENT ACCESS
Fianna Fail Senator Teresa Costello told the Irish Sun that she has written to the Health Minister to ask her to intervene with the situation and speed up access to the medicine.
She said: “It is out of the government’s hands at the moment as the manufacturer has to put in their application and I’ve engaged with the drug company and they said they are going to apply to the HSE so that should be in for July.
“I’ve written to the Health Minister to ask that this drug is made available through an early access programme because these families can’t wait that average time of over 600 days for approval.”
APPROVAL WAIT
The HSE today told the Irish Sun that they are aware of the plight of patients with Duchenne Muscular Dystrophy and are waiting on the drug company to apply for approval here.
They said: “The company must now apply to the HSE for the treatment to be reimbursed through the public health system.
“If the company, Italfarmaco SpA, chooses to submit a pricing and reimbursement application, the HSE will give fair consideration to the application with consideration for the criteria detailed in the 2013 Act.”
The Irish Sun contacted the Minister for Health and the drug company Italfarmco SpA for comment.
