Friday 6 December 2019

Young patients overjoyed as HSE approves 'miracle drug' to treat rare disease

Sam Bailey’s family have been campaigning for Spinraza, which helped Lara McHugh.
Sam Bailey’s family have been campaigning for Spinraza, which helped Lara McHugh.

Parents of children with a rare degenerative illness say they're "over the moon" that the HSE will fund a "miracle drug" that will ultimately save their lives.

The HSE has announced that it will fund the drug Spinraza, the only treatment available for patients with the rare muscle-wasting disease spinal muscular atrophy (SMA).

The life expectancy of babies with SMA type 1 is only eight months, with 80pc dying before the age of one.

It is estimated that there are 25 children in Ireland with SMA types 1, 2 or 3.


The move to fund the drug for patients under the age of 18 has been met with joy by parents and patients, marking the end of a long campaign.

Nine-year-old Sam Bailey, from Rathcoole, Co Dublin, is just one of the affected children who hopes to benefit from Spinraza.

In February, the Herald highlighted how the brave Dubliner was diagnosed with SMA at just 14 months, requiring round-the-clock care since.

His mum Fiona said at the time that whenever her son was admitted to hospital, she feared he would never return home.

Yesterday's news came as a huge relief for the family, who celebrated the announcement by throwing a party for Sam.

"It's such a wonderful day for so many families in Ireland," said his aunt, Jackie Geraghty.

"Thankfully, the HSE and Government have come to their senses and finally approved this miracle of a drug.

"It's been a long, hard battle for Sam, but he's a fighter who has never let his illness define him. He actually said today is the best day of his life," she said.

The parents of a little girl with SMA who was given just weeks to live in 2017 told the Herald that their daughter has made a drastic improvement since Spinraza was made available to her.

Two-year-old Lara McHugh from Blanchardstown was offered a second chance at life when pharmaceutical company Biogen gave Temple Street Children's Hospital a chance to take part in its expanded access programme for the drug.

Father Niall McHugh told the Herald that Lara can now fight off her own illnesses without needing to go to hospital.

"Last year, any time she picked up a minor cold she would have to spend up to two weeks in hospital," he said.

"Now, thanks to Spinraza, her immune system kicks in whenever she gets sick, which means a common cold won't lead to anything more serious.

"It's such a relief that this drug is now going to be funded for children all over Ireland, because for us it's made a huge difference," he said.

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