'It's a travesty' - Adults with rare degenerative illness accuse HSE of leaving them to suffer
Adults with a rare degenerative illness say they’re being left to suffer after the HSE excluded them from getting a “miracle drug”.
This week, the HSE announced it will fund Spinraza – the only treatment for people with rare muscle-wasting disease spinal muscular atrophy (SMA) – but only for patients under 18.
The move has been met with joy by parents of the 25 children in Ireland living with the condition.
However, the SMA Ireland charity said the decision feels like a “hollow victory”, stating that the estimated 25 adults suffering from the disease have been let down.
Jonathan O’Grady (45), who has SMA type two, told Independent.ie that without this life-saving treatment his condition will continue to get worse.
“It’s a travesty,” he said, speaking from his home in Dundrum.
“I would deem myself as a very fit person, but over the years it’s clear that my condition is getting worse and worse.
“When I was in Trinity College, I could dress myself from my wheelchair. But now I can’t even lift my hands to eat.
“I’m still the same person I always was, but I’m getting worse, and without the benefits of Spinraza I don’t know where I’ll be in 10 years’ time.”
Mr O’Grady described the HSE’s decision to fund the drug to children as “bittersweet”.
“It’s fantastic for the children with SMA who have been granted new hope for their future, but we just don’t think it’s very fair that the HSE have excluded adults,” he said.
Secondary school teacher Mary O’Sullivan (50), from Co Kerry, is another adult who suffers from SMA (type three).
Ms O’Sullivan said she was heartbroken when she told her daughter that she wouldn’t have access to Spinraza. “I was just really devastated,” she said.
“On the one hand I was over the moon for the kids, but breaking the news to my daughter on the way home from school that I wasn’t entitled to this drug was really heartbreaking.
“We put our hearts and souls into this campaign and it’s just such a pity that people like me are being excluded from this miraculous treatment.”
Ms O’Sullivan, who is also a breast cancer survivor, said she is able to get around with the use of a walking stick, but is “very worried” about the future.
“This drug stops the progression of SMA and in some cases even reverses some of the mobility problems people like me have,” she said.
“Part of me wants to give up, but my stronger side knows that this fight is long from over. I owe it to my family and everyone else who’s part of this campaign.”
A HSE spokesperson said the rare diseases committee’s recommendation was “clearly targeted at the youngest and most severely affected SMA patients, and this group is the clear priority for the HSE”.
“The actual patient assessment and approval process will be the means for determining access on an individual case-by-case basis.”
SMA Ireland said it accepts there must be some criteria for access to the life-changing drug.
But it described the “arbitrary age cap” as “a crude method with little scientific support”.
When the request for availability was originally turned down, the HSE said the drug would cost about €600,000 in the first year to treat each Irish child with the disease and €380,000 a year afterwards.