Kiwi Mum pleads with Pharmac to fund life-changing drug Spinraza for her two children

The mother of two young boys with a rare disease is begging Pharmac to fund a medication which could change her children's lives.

Lani McLeod's two sons, two-year-old Lincoln and 11-week-old Harlan, both have spinal muscular atrophy (SMA) - a neuromuscular disease which can rob a human of their most basic functions such as sitting, standing, and even breathing. 

The one drug which could change her children's lives, Spinraza, is unfunded.

McLeod says funding Spinraza for all SMA affected children would cost $6 million annually - but despite a strong funding recommendation to Pharmac from the Rare Disorders Subcommittee it's no closer to being available. 

Pharmac chief executive Sarah Fitt said the recommendation is not a guarantee. 

"We are continuing to work with the supplier of Spinraza on their funding application and will consider any further comment or information they provide.

"Having a fixed budget means we need to make careful and considered funding choices in the interests of all New Zealanders."

McLeod says she doesn't think she's asking for much.

"For our boys and the rest of the children it basically stops the disease in its tracks and allows them to gain a bit more function with what they haven't lost yet - so the earlier the better."

McLeod's partner Jim says funding would be "huge".

"At the moment, trying to get Lincoln around, he's at the age where he wants to be exploring but he just can't - he's stuck with what he's got in front of him because he relies on other people to move him around," he said.

The couple is also desperately trying to get a wheelchair for Lincoln, so he can have more agency.

It's been a two-month wait, just to be put on a waiting list.

"He's getting very hard to carry around - we've been informed we're on the waiting list, but that's coming up on three months just for a waiting list - now we have to wait for it to be approved."

Wheelchair provider Enable said due to COVID-19 there are 21 people in line before him for a chair.