13-year wait and counting . . .
Diagnosed with spinal muscular atrophy at 15 months old, the odds have been stacked against Stella Beswick, with a medical opinion that she would be ‘‘lucky to make it out of childhood’’.
Fast-forward to today and looking at Stella, now 14 years old, most would not see a sick child but the reality, her mum Emily said, was that without access to expensive, non-funded drugs, a sharp decline was inevitable. As her mum spoke about having to consider an end-oflife plan for her daughter, sitting among submitters and in front of MPs was Stella, listening intently, on behalf of those, like her, with spinal muscular atrophy.
Hope has sustained the Beswick family, with each year bringing more scientific advances and eventually treatment options. ‘‘Now that a game-changing treatment [Spinraza] is finally being made available, the thought that it is inaccessible for Stella is utterly devastating,’’ Emily said.
In May, Pharmac deferred its decision on whether to fund the drug while awaiting the outcome of two clinical trials – expected by the end of the year. The advisory group cited Spinraza’s high costs against a lack of data to prove its long-term efficacy.
With Stella having been in a wheelchair from age 2, suffering poor sleep and severe scoliosis, making her wait any longer for the drug was incomprehensible to her mum. ‘‘We’ve been waiting 13 years for a treatment to arrive and now it is finally here, it is out of our reach.’’