Monday 24 April 2017

Baby Lara gets gift of life thanks to 'wonder drug'

Four-month-old Lara McHugh with her father Niall McHugh and her mother Celine Power,
who cried with joy when they got the good news
Four-month-old Lara McHugh with her father Niall McHugh and her mother Celine Power, who cried with joy when they got the good news

Ian Begley

A baby girl given just a few weeks to live has received the gift of life following the approval of a new "miracle drug".

The parents of four-month-old Lara McHugh said they "cried with joy" when they received the good news that their daughter could avail of the Spinal Muscular Atrophy (SMA) drug Spinraza.

"We're absolutely delighted," her father Niall said. "My fiancée and I were jumping around the house crying with joy. How can you contain yourself when you hear news like that? Without this drug Lara would definitely die within only a short space of time, but now there's hope."

Baby Lara, from Blanchardstown, was diagnosed with rare condition Type 1 SMA just six weeks after she was born.

The genetic muscular disease makes Lara's muscles extremely weak. A common cold can easily turn into pneumonia which can take the lives of children.

Lara and another 22-month-old baby girl from Dublin are the only two infants in the country with the condition.

Four-month-old Lara McHugh
Four-month-old Lara McHugh

On Wednesday, pharmaceutical company Biogen gave her a second chance at life by offering Temple Street Children's Hospital a chance to take part in its expanded access programme for "wonder drug" Spinraza.

In 50pc of cases it can stop the advancement of SMA1 and in 40pc of cases, it reverses symptoms. It has been very well received in the US, but has yet to be approved in the EU.

"Our whole aim was to get access to this treatment on a compassionate basis, which Biogen has now done. This means we have immediate access and don't have to pay for it," her father said.

"Temple Street has ordered the drugs which will take a couple of weeks to arrive.

"There's still the likelihood that Lara will be in a wheelchair and prone to infections, but this is stuff you can manage and live with."

Irish Independent

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