THE mum of a Cork boy who has spinal muscular atrophy (SMA) has described a decision not to approve the Spinraza drug as “an absolute heartbreak”.
It has emerged that the HSE rejected the latest costing by Spinraza producers Biogen for the drug last Thursday — just days after the NHS in the UK approved it for England and Wales.
Youghal mum Rebecca Bulman is hoping her son Harry and up to 25 other children will have better news in the coming days, after a fresh consultation between Biogen and the HSE.
She said: “We are waiting for a final answer in early June.”
Her five-year-old son Harry was diagnosed with SMA when he was 18 months old. He has to use a wheelchair and suffers from scoliosis as part of his condition.
In February, the HSE wrote to Biogen, the producers of Spinraza, to say the agency was refusing to provide reimbursement of the drug at its current price.
Following the February decision, parents, their children and supporters organised a demonstration at the Dáil, and a petition was signed by up to 90,000 people.
That demonstration was held on Rare Diseases Day.
Harry was diagnosed with SMA in February, 2015. His family heard about Spinraza two years later and were hoping that he could have access to it, but it is not approved in Ireland.
Rebecca said his scoliosis is getting worse . He finds it difficult to write because his hands are weak because of the condition.
SMA reduces a person’s physical strength by affecting the motor nerve cells in the spinal cord. This can affect a person’s ability to walk, eat or breathe.
Little Harry recently picked up a cold which lasted for two months because he could not fight it off.
He attends school in Bunscoil Mhuire in Youghal, and also attends Enable Ireland regularly for therapy.
In announcing its decision last week, NHS England said: “While not a cure, trials have shown that nusinersen (Spinraza) can slow the effects of SMA in some cases, allowing babies and toddlers to develop stronger muscles and survive for longer without breathing support.”
The drug will now be administered to patients who their clinicians believe will benefit from it.