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'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.

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

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.

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“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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