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The Taoiseach said he would find out why children were being denied treatment. Shutterstock/chuanpis

'I've can't lift my arms, brush my hair, or even go to the toilet alone': Children still waiting on Spinraza drug, Dáil told

The drug is used to treat SMA which can develop in babies and toddlers and can significantly reduce life expectancy.

A NUMBER OF children are still waiting on access to Spinraza, an approved treatment for children with spinal muscular atrophy (SMA), the Dáil heard today.

SMA is a rare, debilitating and life-threatening disease that affects nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing. 

Where it develops in babies and toddlers it can significantly reduce life expectancy. While it is not a cure, trials have shown that Spinraza, also known as nusinersen, can slow the effects of SMA in some cases.

Last year, the then Minister for Health Simon Harris announced that the HSE would fund the drug for children only.  

Speaking in the Dáil today, Independent TD Joan Collins said 12 children are still waiting to commence treatment.

One of those waiting is a 17-year-old woman. Her father, Pat, sent Collins an email, which she read into the Dáil record today.

In the email, the father included a personal statement from the young woman waiting on the drug.

“Each day my muscles and body get weaker. When I was 12, I lost my ability to walk and by now, at 17, I have lost the ability to lift my arms up over my shoulders, brush my hair, turn myself over at night and even go to the toilet and have a shower alone. I need assistance to do every little thing.

“I have lost so much quality in my life that I know Spinraza could bring back. Since my muscles have become weaker, I suffer from extremely bad anxiety. It has gotten to the stage that I cannot leave my house without my mam being by my side and I am so anxious that I am not able to sleep alone in my own room downstairs without panicking.”

Collins told the Taoiseach Micheál Martin that the woman’s neurologist was in contact with the HSE before the pandemic and was told no beds were available and the required care cannot be followed up.

“This is why the young woman has not been able to access Spinraza,” she said, stating that the families of the 12 children waiting on the treatment have not been told what is happening.

“I will certainly do what I can to find out what is going on with those 12 children, and particularly the 17-year-old young woman referred to by the Deputy. I remember the campaign and the major commitment made by the families involved.

“There is an ongoing issue with orphan drugs, or drugs that are developed for what are classed as small populations in particular disease categories. I do not know why 12 children remain to be treated right now but I will follow up on it and see what we can do to facilitate access to the drug,” said the Taoiseach.

“I will certainly do what I can to get to the bottom of this,” he said, stating that every day matters to families in cases like this.

“It is a small number of people who need this treatment urgently,” said Collins.

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    Mute Lewis Armstrong
    Favourite Lewis Armstrong
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    Jan 14th 2024, 12:52 AM

    Great achievements and still so young but leaving his partner and kid to party and live the Hollywood life is unfortunately where he will never grace the echelons of Cillian Murphy who is all class

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    Mute Chris
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    Jan 13th 2024, 10:57 PM

    I’m sorry but his acting is pretty similar no matter the character, so his acting register must improve or….I really don’t know why there is a buzz about him, maybe his appearance?

    177
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    Mute NoelDublin
    Favourite NoelDublin
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    Jan 13th 2024, 11:43 PM

    @Chris: oh disagree. His performance in Saltburn is show stealing. Very subtle and beautiful. Yes, his looks are distinctive. All the greats were.

    102
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    Mute Leinsterprincess
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    Jan 14th 2024, 10:10 AM

    @NoelDublin: yea I loved all the various accents he was doing throughout the film lol

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    Mute Alan
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    Jan 14th 2024, 1:01 PM

    @Leinsterprincess: thought he looked too old for the part

    5
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    Mute Lapland
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    Jan 14th 2024, 1:26 AM

    Jeez the Irish on here doing what the Irish are good at… slating their own . Shameful comments..

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    Mute Thesaltyurchin
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    Jan 14th 2024, 11:07 AM

    @Lapland: World capital of begrudgery. It’s why we have no services or infrastructure.

    42
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    Mute T S
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    Jan 14th 2024, 1:46 PM

    @Lapland: Begrudgery is unreal isn’t it? The last curse of the Catholic Church.

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    Mute Binne
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    Jan 14th 2024, 8:29 AM

    Barry Keoghan is an amazing talent.. Saltburn is a story of manipulation told in a different way.. Barry performance is out of this world and I wish him the very best, so proud to see this young man carve his way to the very top in the film industry.. as for his personal life it’s none of anyone’s business

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    Mute Furious George - The Wasp
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    Jan 13th 2024, 10:07 PM

    And he got his lad out

    93
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    Mute Colin Howard
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    Jan 14th 2024, 8:37 AM

    It’s good to see that the aul Irish begrudgery is alive and well this morning. A young man who’s had a tough upbringing has made it on the world stage and most on here just want to put him down. But wait, the Brits try to claim him as one of their own? The very same begrudgers will he back on here blowing a gasket over that!!

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    Mute Jack Moss
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    Jan 14th 2024, 2:42 PM

    @Colin Howard: The Brits didn’t try to claim them as their own . GQ magazine is American . Its offices are in New York and its own by an American . An article written in a rag says he is British and every one gets upset .

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    Mute Megan Ward
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    Jan 15th 2024, 12:05 PM

    @Jack Moss: They didn’t even say he was British, they referred to him as ‘one of our greatest’ actors, as in the world. If it was specific to nationality then they’d have been calling him American as it was the American GQ article lifted.

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    Mute Paul Kavanagh
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    Jan 13th 2024, 11:57 PM

    good for him. living the dream.

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    Mute Aidan McCormack
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    Jan 14th 2024, 12:24 AM

    Here am i the only one that thought saltburn was a load of…. you know what i want to say. Nonsense

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    Mute Niall Madden
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    Jan 14th 2024, 7:02 AM

    I know that a few years ago while in Galway filming the Inisherin film he was out on the town with his brother and got a couple of clouts off said brother. He was all over the media telling us how he was attacked in Galway and was terrified for his life. Award winner alright!

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    Mute Benny Mchale
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    Jan 13th 2024, 11:04 PM

    4 out of10. Knew the cat question. Thought he looked like the joker and guessed the other two.

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    Mute Edward Hughes
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    Jan 14th 2024, 12:43 AM

    Got a break in life and making the most of it. He’s flavour of the month( to be crass). First and only pupil from the Colin Farrell school of acting. He’s improving all the time. Last role in salt burn was a hard watch but got there in the end. Hopefully he realises it and works to develop the potential. Leave him to it and see what happens. “ buy Irish”

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    Mute Alan Casey
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    Jan 14th 2024, 2:02 AM

    Ah he’s a cheeky little bollix.

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    Mute nollaig o cellaigh
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    Jan 14th 2024, 12:46 AM

    7 / 10

    Good actor hope he keeps his feet on the ground

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    Mute Alan Casey
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    Jan 14th 2024, 2:04 AM

    @nollaig o cellaigh: Apparently not off to a good start in that regard. Dumped his girlfriend and child.

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    Mute Patrick O'Leary
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    Jan 14th 2024, 11:47 AM

    Never heard of him so guess “Not a lot” would be the answer.

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    Mute Bass Boss
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    Jan 13th 2024, 10:46 PM

    no artikel over the journoz killed in kiev?!!.. navalny le traître..

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    Mute Rover
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    Jan 14th 2024, 12:48 AM

    Made up here that he cut his teeth in Fair City

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    Mute Paul whitehead
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    Jan 14th 2024, 12:50 PM

    Typical of Irish begrudgery. Knocking our own .. Didn’t he get his lad out??

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    Mute HurdyGurdyMan
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    Jan 14th 2024, 8:18 PM

    Great to see the lad doing so well

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    Mute Michael Fitzpatrick
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    Jan 14th 2024, 2:01 PM

    He’s NOT BRITISH …..

    FFS !!! When will our “so called” nearest neighbours ever learn to STOP laying claim to things that AREN’T theirs ……

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