Hastings mother Rachel Shaw says medicine to help her two oldest children exists, but is out of reach. From left are children Skylah-Rose Shaw, Chloe Matthews and Zayden Shaw in 2020. Photo / NZME
Hastings mother Rachel Shaw says medicine to help her two oldest children exists, but is out of reach. From left are children Skylah-Rose Shaw, Chloe Matthews and Zayden Shaw in 2020. Photo / NZME
Parents of local children affected by spinal muscular atrophy say approval of a new medicine has not done much to help with still no Pharmac funding.
Eleven-year-old Liam, a Hastings boy who has the rare disorder, will be entering the biggest surgery he has ever had possibly as soon asAugust, to get spinal rods put in and a fusion.
His mum Stacey Reading said there was a risk the already lengthy surgery would have to be split into two if Liam's lungs don't hold up.
"Knowing there is something out there that can help him and we can't get it... it's really, really heartbreaking".
New Zealand has two medicines approved for the treatment of SMA, Spinraza and more recently Evrysdi (risdiplam), which was approved earlier this year.
Although Spinraza has been on the waiting list since 2018 and has been listed as the highest priority since about 2020, neither it nor Evrysdi have been approved for funding by Pharmac.
In Australia they started funding Spinraza in about 2018 and have also begun funding Evrysdi and another SMA treatment, Zolgensma, since then.
"If you are born in New Zealand with a rare disorder, they have left you to die, that's it. They don't care about you at all."
Pausing, Reading said it's hard for her to talk about Liam's situation without tearing up.
11-year-old Liam, a Hastings boy with Spinal Muscular Atrophy, will be going into his biggest surgery ever soon to get spinal rods put in. Photo from 2020. Photo / NZME
Hastings resident Rachel Shaw is a mother of three children, including two with SMA, Skylah-Rose, 10, and Zayden, 12.
She said the winter bugs have kept her kids home sick a lot recently and their conditions were worsening without treatment.
"With medication, we wouldn't be needing so many appointments and new equipment and doctor's visits."
Zayden needs assistance eating and Skylah-Rose has lost the ability to walk distances due to the pain it puts her in.
"Everyday life is hard when you know there is something out there that can help quality of life and we just can't get it."
A recent independent review of Pharmac found that people with rare disorders have faced disproportionately negative health outcomes due to systemic failings.
Shaw is convinced the review findings won't lead to meaningful change for her and her children.
"They're not interested in making a difference, they just want to look like they're doing something."
Reading wasn't holding out hope on any change coming on the back of the report.
"They might have that written in front of them, but that still doesn't mean they're going to do anything about it."
Fiona Tolich, SMA patient advocate and someone who has the disorder, said patients in New Zealand can't get access to medicine without Pharmac funding because nearly all insurance wouldn't cover rare disease medicines.
"In New Zealand, children die on average at 13 months old from a very treatable disease. It's appalling".
She said over 25 per cent of kids with SMA have left New Zealand permanently for treatment.
