DRUG FIGHT Desperate parents of toddler with rare muscle disease battling to have life-extending wonder drug approved in Scotland

Little Zac Cameron lives with spinal muscular atrophy (SMA), a devastating condition which leaves babies disabled and often with a life expectancy of little more than two years

THE parents of a toddler with a rare muscle disease are battling to have a life-extending wonder drug approved in Scotland.

Little Zac Cameron lives with spinal muscular atrophy (SMA), a devastating condition which leaves babies disabled and often with a life expectancy of little more than two years

Although there is no cure, his mum Amy, 30, said Zac’s life has been transformed by a miracle treatment named Spinraza. Thanks to the treatment, Zac is approaching his third birthday June.

Amy, from Alloa, is now fighting to have the drug approved in Scotland after Zac was chosen for a trial.

The pharmacy assistant said: “Zac is receiving the treatment under an access programme, so he’s been on that for almost a full year.

“He’s had massive progress and is now able to hold his head up most of the day and be able to play, sit at the table and communicate.

Zac has been fighting the disease since birth
“He can now lift up his cup and feed himself, he can splash in the bath, he can kick and he can interact more.

“When Zac was diagnosed the medical team said they felt he would be a candidate and asked if we’d like to get him into the programme.

“While it’s not a cure, it is buying us valuable time with Zac and enabling him to reach milestones we never thought would be possible, such as picking up his cup for a drink and moving his legs.”

Zac was diagnosed with SMA Type 1 — the most severe form — at 17 months in November 2017, leaving Amy and accountant dad Craig, 31, devastated.

The condition leads to the gradual loss of the ability to walk, crawl, move, swallow and even breathe.

Amy first noticed symptoms when Zac was eight months old. She said: “It was devastating because it’s a life-limiting condition.

“However, our outlook was that we would give him the best we could and spend as much time with him as possible.”

The UK-wide “compassionate access scheme” gives only children with SMA Type 1 free access to Spinraza while it is being approved.

Now his parents are calling on the Scottish Medicines Consortium (SMC) to approve it in Scotland.

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On March 13, the SMC held a Patient and Clinical Experts meeting to hear the case.

Next month will give families and charities a chance to make final submissions.

Amy added: “It’s given us massive hope for the future. From what we’ve seen with Zac everyone who is affected by SMA has to get this treatment.”
lisa.boyle@the-sun.co.uk

 

Source: https://www.thescottishsun.co.uk/news/2473636/zac-cameron-parents-spinal-muscular-atrophy-spinraza-miracle-drug/

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