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'It's life or death for these kids' - parents want 'right decision' from HSE over drug

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Celine Power and Niall McHugh with daughter Lara.  Picture Credit:Frank McGrath

Celine Power and Niall McHugh with daughter Lara. Picture Credit:Frank McGrath

Celine Power and Niall McHugh with daughter Lara. Picture Credit:Frank McGrath

A little girl who was given only weeks to live last year has made a drastic improvement thanks to a new "miracle drug" from America which the HSE still has not approved.

Two-year-old Lara McHugh, from Blanchardstown, was diagnosed with an extremely rare condition known as Type 1 Spinal Muscular Atrophy (SMA) when she was six-weeks-old.

The life expectancy of babies with SMA is only eight months, with 80pc dying before the age of one and most of the rest dying before the age of two.

The Herald spoke last year to Lara's heartbroken father, Niall, who feared his daugh- ter, who was four-months-old at the time, would not live past Easter.

However, US pharmaceutical company Biogen gave her a second chance at life by offering her a chance to take part in its expanded access programme for "wonder drug" Spinraza.

Fight

Now, more than a year later, Lara can move, fight infections and is even starting to talk.

"She's a totally different child and wouldn't be here at all without this drug," said Mr McHugh.

"When she was first diagnosed, she lost all motor functions and couldn't move at all.

"She was getting progressively worse, and if she had caught something as simple as a cold she would have died.

"Since starting her treatment, we've seen enormous improvements. She's doing her wheelchair training now, turning pages in her books and can hold her head up - she's a totally different child.

"She has even said her own name a few times and other words like 'mama'.

"Lara was also in a very bad way earlier this year with respiratory problems, but her doctors informed us she managed to fight off the infection herself, which was just incredible."

Since the HSE is currently in negotiations with Biogen, the expanded access programme for Spinraza has stopped.

This means that any child born with SMA at the moment will not be given access to the drug.

Mr McHugh was assured, however, that anyone currently on the expanded access programme, including Lara, will not be affected.

It is understood a decision will be made on Friday on whether the expensive drug will be reimbursed in Ireland.

Only last Thursday, families, supporters and sufferers of SMA gathered outside the Dail to call for access to Spinraza.

The McHughs have seen first-hand the benefits of the drug and how the earlier a child receives it, the better their long-term prognosis will be.

"The longer kids are without treatment, the worse they'll be," said Mr McHugh.

Essential

"The drug only works before the symptoms of the condition start. This means that kids with SMA are losing the ability to walk because the effects can't be reversed.

"It's essential and so important that the HSE makes the right decision - it's truly life and death for these kids."

The disease makes muscles extremely weak, especially in the legs, upper arms and neck.

A common cold can easily turn into pneumonia, which is what usually takes the lives of affected children, along with respiratory failure when they no longer have the lung or chest muscles to be able to breathe on their own.


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