Pharmac decision on SMA drug lifts ‘underlying guilt’

Lani McLeod has had an underlying feeling of guilt for two years that her boys had access to life-changing treatment others did not. That changed this week.

Her sons Lincoln, 4, and Harlan, 2, have spinal muscular atrophy (SMA) type 2 – a type of the motor neurone disease – but were fortunate to be granted compassionate access to risdiplam, a drug to treat SMA, since March 2021.

Pharmac announced this week it will begin consultation on funding another SMA treatment, nusinren, the drug branded as Spinraza, to roll it out for people aged 18 and under from January 1.

‘‘It’s so exciting knowing it’s not just us now as there has been that underlying guilt with the happiness of getting [treatment] that ... [many others were in need] and there was nothing we could do,’’ McLeod said. ‘‘It will make things easier for families of any newly diagnosed kids and take the stress of considering moving countries or fighting for treatment away.’’

The shock of an SMA diagnosis was bad enough, McLeod said, without the heartbreak of being told there was a treatment available but it was out of reach. ‘‘For a newly diagnosed parent, it’s still going to be extremely tough but having the treatment available will take a huge weight off. The faster you have access to it the better. In our case treatment early would have saved a lot of heartache and a lot of struggles for the boys and us.’’ Everything changed when the brothers were granted compassionate access to risdiplam as McLeod and her husband James started seeing their boys ‘‘stop declining and making gains’’.

‘‘Within weeks we noticed the difference. The boys are making some amazing progress and haven’t had any further loss of motor skills.’’

Lincoln is benefiting from an improved immune system, meaning fewer trips to the hospital for minor coughs and colds, and making physical gains.

Before the treatment, the then 8-monthold Harlan was struggling to lift his head off the ground but now ‘‘he’s off crawling, climbing up on furniture and getting into mischief’’.

‘‘It’s regular kid stuff that he never would have had the chance to do if he didn’t have access to the medication. So it’s an awesome feeling to know other children will have a chance at a regular childhood – as regular as it can be with SMA.’’