Friday 26 May 2017

Baby girl given 'gift of life' after 'miracle drug' made available to Temple Street Children's Hospital

Lara McHugh
Lara McHugh

Ian Begley

A baby girl given just a few weeks to live has been given 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,” said her father Niall. My finance 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 an ultra rare condition known as Type 1 SMA just six weeks after she was born.

The genetic muscular disease makes Lara’s muscles extremely weak, with the weakest muscles being in her 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.

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

On Wednesday, Pharmaceutical company Biogen gave her a second chance at life by offering Temple Street Children’s Hospital a change to take part in it’s expanded access programme for their “wonder drug” Spinraza.

In 50 per cent of cases it can stop the advancement of SMA1 and in 40 per cent of cases, it reverses the symptoms.  The drug has 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 that we have immediate access to the treatment and don’t have to pay for it.

“Temple Street have ordered the drugs which will take a couple of weeks to arrive, but as soon as they do they will be administered to Lara.

“We still have to hope that she responds well to the treatment with further hopes that it will stop the progression of the SMA disease.”

Spinraza helps to boost protein production in an affected infants body where there was none before. Babies who have previously benefited from the drug have regained their motor skills, but to date there is no definite cure for SMA.

“There are still the likely hood that Lara will be in a wheelchair and prone to infections, but this is stuff you can manage and live with,” added Niall.

“This treatment has just become available and who maybe they’ll find a cure for the disease within the next five to 10 years.”

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