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Monday 24 July 2017

'We thought little Lara only needed physio... but she is fighting for her life'

Parents wait for approval of drug as baby battles for life

Lara McHugh
Lara McHugh

Ian Begley

A baby girl could have mere months to live while her family faces a desperate wait for a new "life-enhancing" drug to be approved.

Four-month-old Lara McHugh from Blanchardstown was diagnosed with an ultra rare condition known as Type 1 Spinal Muscular Atrophy (SMA) when she was six weeks old.

The genetic disease makes Lara's 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 children affected, along with "respiratory failure" when they no longer have the lung or chest muscles to be able to breathe on their own.

The life expectancy of babies with SMA Type 1 is only eight months, with 80pc dying before the age of one, and most of the rest dying before two.

There are currently just two babies with SMA in Ireland - both based in Dublin.

Niall McHugh said his world collapsed as soon as his baby was diagnosed with the condition.

"My fiancée Celine and I were absolutely devastated when we heard the news. We were in complete shock when we were told how serious our daughter's condition was and are just plagued with grief.

"We brought Lara in to Temple Street's Children's hospital to have a check-up when we noticed her legs weren't moving as well as the doctors expected.

"We thought she would just need a bit of physio. After many tests, doctors eventually diagnosed our daughter with SMA."

Niall added that there is a ray of hope for his daughter due to a new drug for SMA patients recently given Food and Drug Administration (FDA) approval.

Biotech Company Biogen, which produces the drug Nusinersen, saw positive results during clinical trials in the US. However, the drug has not been given authorisation as of yet from the European Medicines Agency (EMA).

"We're hopeful that the HSE and the National Centre for Pharmaeconomics (NCPE) can approve the treatment Lara needs so she can take part in Biogen's expanded access programme.

"We're not sure if this drug could save our daughter's life in the long-run, but it will certainly extend her life."

The HSE said it is aware that Biogen applied to the EMA for market authorisation in the EU.

"The HSE understands that application to EMA was submitted in September-October. EMA assessment processes generally take around one year.

"It is unlikely that the HSE will receive a pricing and reimbursement application until later this year."

Irish Independent

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