Through this agreement, people with SMA types 1, 2 and 3 can now access the treatment, although there are some restrictions attached. This followed the treatment becoming available in Scotland and roll-out is beginning in Wales and Northern Ireland.
While we know that not everyone who can benefit from the treatment is receiving it – and we are working hard to change this – the announcement last summer was good news for Mark Wilson. His son, Aadi, has SMA Type 2.
Mark remembers the moment he heard Spinraza was to be made available through the NHS.
“I had woken up early for work, turned my phone on, and saw an email breaking the news,” he says. “My wife, Panna, who was pregnant at the time, was still fast asleep, but I had to tell her.
“We then immediately texted our family and friends with the news, and once I got to the school where I work my friends had plastered the door to my classroom with messages of congratulations.”
“We were overjoyed.”
For families like Aadi’s, Spinraza represents hope. “It means we can feel more excited about what the future might hold…we don’t know how much impact it will have on our son, we just want the opportunity to try,” says Mark.
“We’re lucky to have such a beautiful son, and he deserves the very best.”
The family joined MDUK, SMA charities, clinicians and the wider SMA community in campaigning for Spinraza to be made available in what was a long, and often emotional, process. It took 18 months for NICE to approve the drug.
“The delays have been incredibly frustrating and completely unnecessary, and when we recount to our friends the story so far…they can’t believe how slow the whole process is,” says Mark.
Aadi has to travel to Sheffield for treatment as his local NHS trust in Leicester cannot deliver it.
“We will do everything to give him the greatest life we can.”
Mark says, “Aadi keeps us positive, and like him we face every challenge with a determination and a smile.”