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More on KentOnline
The mother of a teenager with a rare muscle disease says the decision to make a drug that treats the condition available on the NHS is a dream come true.
Mel Bolt, from Minster, has been campaigning for Spinraza to be made available through the health service for about 18 months to help her daughter, Abbie, who was diagnosed with spinal muscular atrophy (SMA) when she was two years old.
The former Westlands School pupil, now 15, has since battled with the incurable muscle-weakening condition and is a full-time wheelchair user.
Health bosses originally rejected the drug, saying the long-term benefits were “highly uncertain” and the cost was “too high for it to be considered a cost-effective use of NHS resources”.
But, last Tuesday, the National Institute for Health and Care Excellence (NICE) announced it would reverse its decision and recommend Spinraza – also known as nusinersen – as standard treatment for adults and children who are living with SMA.
“It’s a dream come true,” Mel said. “Knowing the drug will not only benefit Abbie but other children and adults too is amazing.”
The mother-of-two, who teamed up with TreatSMA, a community of people affected by SMA, to fight for the treatment, said all the hard work had paid off.
“It’s been a long 18 months, filled with uncertainty and worry, but it’s all been worthwhile,” Mel said.
“I started crying as soon as I heard the news – it just hit me that we had been listened to and this was happening.”
The 33-year-old said if Abbie could have access to the drug, it may help her become stronger and reach milestones she hasn’t yet been able to.
“Abbie can’t stand, walk, she can’t lift her hands up to brush her hair. Even if it just gives her the strength of her arms – for her it’s about doing the normal things a 15-year-old wants to do.”
Mel said she thought it would be a few months before Abbie would receive the treatment.
“If it works, Abbie is most excited about being more independent and doing things we all take for granted, like going out with friends and picking her drink up for herself.”
