For the first time in her life, two-year-old Charlotte Bond is showing signs that one day she could walk on her own.
It's a proud feeling for her mother Kristie, who uprooted the family's life in Auckland's Pukekohe to access a life-saving drug in Australia.
Charlotte's motor-neuron disease - spinal muscular atrophy - causes basic movements to be exhausting as muscles around her body slowly die.
Kristie says she's felt forced out of the place she's called home as Pharmac, the company who approve the drug for public funding stall access to Spinraza.
"A lot of people think so much of New Zealand, but the health system is not just letting us down but a lot of people," she told Newshub.
The mother-of-two is now an Australian resident and Charlotte can be treated with Spinraza through her Medicare card as the drug is funded under the public health system.
Kristie, her son and Charlote have settled into a home on the Gold Coast. Her partner, the children's father, goes back and forth as continues to run his business. The move has been a huge financial strain on their family who have accepted help from loved ones.
Spinraza is in 45 countries, including Australia, where it has been listed on the Pharmaceutical Benefits Scheme since June 1, 2018.
It has been proven to drastically slow down, even stop, the progression of SMA which could help about - 35 New Zealand children with this condition.
Now after four doses of her 'hulk juice', Charlotte is achieving more, feeling stronger and pushing herself
Leaving their close-knit family behind has been difficult but she hasn't thought twice seeing the differences it makes to her daughter's life.
"I wouldn't change anything now," Kristie says.
She has been blown away by Charlotte's improvements. Reaching above her head would be a struggle with, now she's putting her arms up freely, Kristie says.
"After two doses, I put her in her own bed, she slept all through the night and she's been able to roll over by herself now, which is huge."
Before the treatment, Kristie would help Charlotte turn during her sleep.
Lead advocate for SMA Fiona Tolich, who lives with Type 3-4, told Newshub believes the lack of funding for Spinraza is "our national shame".
"This is a motor neuron disease that is robbing people of their lives and there is a proven treatment to stop it.
"We need David Clark to recognise that under the current way of thinking, rare disorders patients will continue to suffer at the demise of their condition, in some cases like SMA completely unnecessarily."
Pharmac's Rare Disorders Subcommittee considered a funding application for Spinraza in November 2018.
It deferred making a recommendation, asking for the application to be assessed again, once more evidence from two clinical trials is published.
Additional information is expected to be considered after the subcommittee meets later this month.
Health Minister Dr David Clark told Newshub the Government's hands are tied.
"I absolutely understand why New Zealanders suffering from spinal muscular atrophy and their families want affordable publicly funded access to the latest treatments for this disease.
"However, the Government respects the independence and impartiality of Pharmac.
"It is not for us as politicians to second guess the experts at Pharmac about which drugs it purchases."
Biogen, the manufacturer of Spinraza, told Newshub that it is disappointed in Pharmac's handling of the drug for the treatment of New Zealanders with Spinal Muscular Atrophy.
"It is hoped that a swift, positive outcome can be reached with Pharmac to enable those patients who, in consultation with their clinician, can benefit from Spinraza have access to this medicine."
Director of operations at Pharmac, Lisa Williams, understands that people living with rare disorders in New Zealand face challenges and frustrations accessing suitable health care, including access to effective medicines.
She says they have made improvements around decision-making processes to increase funded access to medicines and since 2017/18 have funded over 100 medicines that helped more than 340 people with rare disorders.
Unlike other medicines, she says Medsafe approval is required before Pharmac will fund the medicine.
For people like Tolich "delays are not good enough". As SMA patients continue to wait, it's likely to be another five months before an update is made.
"In that time, children will lose the ability to walk, sit, feed themselves or even breathe," Tolich says.
She believes six years worth of data and the 8500 patients using Spinraza around the world should be proof enough.
"The system is meant to be there to protect us and it’s not. Our Minister of Health needs to recognise his role in being there to serve the people."
To help support Charlotte's therapy and activities click here.
Newshub.
