A 15,000-strong petition calling on Pharmac to fund a drug to treat spinal muscular atrophy is being presented to Parliament as the Auckland family behind it watch their daughter's health deteriorate.
National MP Paula Bennett is presenting the petition - written by mother Kristie Yeoman and grandmother Janine Yeoman - on May 1. Bennett met Kristie and her 2-year-old daughter Charlotte Bond, who has SMA, last month.
Bennett met Kristie and her 2-year-old daughter Charlotte Bond, who has SMA, last month.
SMA is a degenerative disease that affects the control of voluntary muscle movement. It's caused by a loss of motor neurons.
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.
Kristie hopes access to the drug would save Charlotte from an early death.
Last month, 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 - a move Kristie described as "devastating".
Charlotte was diagnosed with type 2 SMA just before her first birthday. She cannot walk unassisted, but uses a pink wheelchair to zip around in.
She is excluded from the very small pool of New Zealanders eligible for Spinraza through Biogen - the American company that makes it.
Biogen provides the drug free of charge to SMA sufferers under the age of 18 - but only those with type 1 SMA; the most aggressive of the three forms of the illness.
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.
Speaking to the Herald on Sunday, Kristie said she'd watched Charlotte's strength fade over recent weeks.
"She hasn't really been crawling as much, she hasn't been able to pull herself up - as she's getting older and she's getting heavier.
"She's started to say, 'mum I can't walk, my legs are broken'.
"I keep saying to her, 'we're going to try to get you some medicine, and it's going to make you walk'."
Kristie, her mother Janine and her partner Rick were trying to stay positive.
Their success in drumming up support for a petition Kristie and Janine launched early last month has provided some hope.
The online petition urging Pharmac to fund Spinraza for all Kiwis battling spinal muscular atrophy had 12,673 signatures before it closed on Tuesday.
Kristie believed signatures gathered from paper petitions she and her mother handed out would bump the total number of signatures up to around 15,000.
The family was "ecstatic" about the reaction.
Support and awareness was built on social media and through their own networks by a community of families affected by SMA.
"There's probably 10,000 people that now know what it is, how rare it is, and how important it is for this drug to be funded."
Bennett has also given her support to the petition, which she said makes a strong case for change.
She described Charlotte as an "absolute sweetheart".
"It is heartbreaking to look at such a gorgeous kid and know they are going to have to go through such a substantial amount of pain in such a short period of time."
Bennett said the Government's downvoting of a select committee inquiry into Pharmac was another blow to the SMA community.
"Part of the issue is how Pharmac is making the decisions, and why."
Meanwhile, Kristie Yeoman was holding out hope funding would be approved and fast-tracked.
If not, Charlotte and her family would likely move to Australia - where the drug was funded.
Kristie said this option was less than ideal - she has a strong support network in New Zealand and doesn't want to take Charlotte away from her family.
"But at the end of the day she needs that drug, wherever that may be."