herald

Thursday 27 April 2017

'Now there's hope' - family's joy as daughter gets 'miracle drug'

Lara McHugh's (4 months) is pictured with her father David McHugh and her mother Celine Power
Lara McHugh's (4 months) is pictured with her father David McHugh and her mother Celine Power

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.

Lara, from Blanchardstown, and 22-month-old baby Nellie Lannon, from Dublin, are the only two infants in the country with the extremely rare condition Type 1 SMA.

"We're absolutely delighted," her father Niall told the Herald.

"My fiancee 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 was diagnosed with Type 1 SMA just six weeks after she was born.

The genetic disease makes her muscles extremely weak, with the weakest being in her legs, upper arms, and neck.

A common cold can easily turn into pneumonia, which is often what leads to the deaths of children.

On Wednesday, pharmaceutical company Biogen gave both families hope by offering Temple Street Children's Hospital a chance to take part in an expanded access programme for Spinraza.

In 50pc of cases it can stop the advancement of SMA1 and, in 40pc of cases, it reverses the symptoms.

"Our whole aim was to get access to this treatment on a compassionate basis, which Biogen has now done," said Niall.

Cure

"This means that we have immediate access to the treatment and don't have to pay for it.

"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 infant's body.

"There is still the likelihood 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 knows, maybe they'll find a cure for the disease within the next five to 10 years."

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