A teenage spinal muscular atrophy sufferer living in Australia is holding out hope Pharmac will fund the drug keeping her disease at bay, so she can move home to New Zealand to study.
Natalie Edwards, 16, has been living in Broome, Australia, for the past six years.
She moved with her mother, stepfather and two stepsisters in 2012 for family reasons, but was given another reason to stay when Spinraza, a life-extending drug for SMA sufferers, became funded through Australia's Pharmaceutical Benefits Scheme last year.
SMA is a degenerative disease caused by a loss of motor neurons that affects the control of voluntary muscle movement. If untreated, the disease makes its sufferer weaker over time before causing an early death.
Spinraza is the only FDA-approved treatment for SMA sufferers. It isn't a cure but has been proven to slow or stop the effects of the disease.
Edwards' plea for Pharmac to fund Spinraza comes just weeks after after a petition, written by the family of a 2-year-old Auckland girl with an aggressive form of SMA, was presented to Parliament by National MP Paula Bennett.
And last week, hundreds of people rallied at Parliament to call for more drugs to be funded by Pharmac.
There are eight petitions calling for 26 treatments to be funded for six diseases, mainly cancers.
Speaking to the Herald on Sunday about the push for funding, Edwards said her future depended on it - as well as the lives of many Kiwi kids.
She wanted to move back to New Zealand, where she grew up, to study engineering at the University of Auckland.
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"Because I am not an Australian citizen I would have to pay overseas student fees if I studied here ... I also would not have access to full disability funding," she said.
"I most likely would not get government funding for a caregiver - which will be highly important when I move out."
Edwards needs help with everyday things like getting ready for school. It takes her mother around an hour to get her up from bed into her wheelchair, then ready for the day.
When Edwards was born she had normal movement. She was diagnosed with SMA at 9 months, at which point crawling was starting to become a struggle.
She stopped crawling altogether when she was just 18 months old.
Doctors didn't expect she ever would walk.
Natalie's mother, Katherine Reeves, said Spinraza had lifted her daughter's quality of life.
"There's small things that she can now do, that she couldn't do before," she said.
"She can turn on a light switch, knock on someone's door and she can speak with a slightly stronger voice. "The biggest thing would be that she hasn't been sick since she's been on it."
Edwards had contracted pneumonia every year, without fail, until she had her first dose of the drug, about a year ago. She got so sick with the illness when aged about six that doctors feared she may die.
Long-term, Spinraza was expected to save Edwards from an early death.
Reeves said getting the drug when her daughter was 15, was "bittersweet".
She had heard of younger children who started the treatment pre-symptomatically when they were just a few weeks or a few months old who developed as a healthy child would.
"They'll sit, they'll crawl, they'll walk. They might struggle to do so, but they're not on all of the stuff that Natalie has to be on."
Natalie needed breathing equipment at night. She was also reliant on her wheelchair.
"She has lost a lot of function that she could have retained if we had had it 10 years earlier."
Earlier this year, Pharmac deferred a decision on funding Spinraza for SMA patients aged 18 and under until the outcomes of two longer-term clinical trials were available.
The deferral was part of the motivation for Christie Yeoman to pen a petition calling on Pharmac to fund Spinraza, to save her 2-year-old daughter Charlotte from an early death.
The petition was presented to Parliament by Paula Bennett on May 1. Reeves' Auckland-based mother Judy was there in support of the cause.
Bennett said she was hugely touched by the plight of SMA sufferers and their families.
"You can't help but be touched by these children and the struggle they have ahead," she said.
The petition will now be assigned to a Select Committee, which would determine whether it goes any further. Bennett expected the committee would take submissions "at the very least".
Talking about her daughter's future, Reeves said she would discourage her from moving to New Zealand - though by the age of 18 it would be her choice to make.
"I feel like she'd be putting her life on the line, effectively."
Spinraza: What you need to know
• Without Government funding, the first year of Spinraza costs around $1 million. Hundreds of thousands of dollars are needed to continue the treatment for subsequent years.
• Spinraza works by causing the body to produce more SMN protein. SMA sufferers have a shortage of this protein, which leads to weakness of the muscles in the shoulders, hips, thighs and upper back.
• Spinraza is the only FDA-approved treatment for SMA sufferers. It isn't a cure but has been proven to slow or stop the effects of the disease.
• Spinraza is given by injection into the lower back. It's given once every four months.