Good news for brave schoolgirl Heidi as SMA treatment begins

She spent 18 months campaigning for the only-known drug to treat her muscle-wasting disease to be approved for use on the NHS.

Now the future is looking brighter after she was given the go-ahead to receive the treatment which her mum Katie hopes will enable her to build up her strength and confidence.

Heidi has spinal muscular atrophy (SMA) - a genetic condition that makes muscles weaker and causes problems with walking and movement.

Although there is no cure, the drug Spinraza, the trade name of the drug nusinersen, can help slow down the effects and help maintain independence.

The National Institute for Health and Care Excellence (NICE) had previously not recommended it due to concerns over its long-term effectiveness and high cost.

Twelve-year-old Heidi and her family joined a national campaign, led by TreatSMA, calling for the drug, which was already available in Scotland, to be funded by the NHS, believing any treatment was better than none.

But when it was finally approved in May last year their joy was short-lived after they realised that Heidi was unlikely to qualify for the drug as she could no longer walk the required five steps.

Fight

The family continued to fight, arguing that Heidi shouldn't miss out as she would have met the criteria had it not been for the delays in getting the drug signed off.

Katie, aged 34, from Bradmore, Wolverhampton, said it was a relief to see Heidi, who now attends St Edmund's Academy, finally start receiving the treatment.

"It's been good for her. She fought for so long for her and others to have the treatment and in that time she went from being able to take a few steps to being able to take none.

"They originally said Spinraza was approved for all SMA types but then said children with type 3, which Heidi has, needed to have walked five steps within the past 12 months.

"It was borderline for Heidi because was just outside that. It didn't seem fair that just a few weeks or months should make a difference," she said.

But it's not been an easy journey for Heidi who had originally been due to start the treatment in October.

It was delayed after she underwent spinal fusion surgery to urgently correct a curve in her spine.

Complications

Due to complications she ended up spending almost three months in New Cross Hospital as her wound took longer to heal that expected.

"It was a rough time for Heidi and she was in theatre six times so they could keep her wound clear of infection. There didn't seem to be any light at the end of the tunnel. It was great when she came out of hospital.

"She missed the first few months of Year 7 but she's just started back at school full-time.

"She was complaining of backache before the surgery but she isn't now. It was a hard time but I think it's been worth it and it's done her the world of good. We're just getting back to normal," said Katie.

They have, however, been left with some good memories of her time in hospital thanks to pop star Ariana Grande.

"When Heidi had her surgery she was supposed to be going to see Ariana in Birmingham but she couldn't go.

"With the help of her fans I got a message to Ariana and she got her assistant to bring a signed jumper to the hospital and Ariana sent her a video message," says Katie.

Heidi has so far received three of four 'loading' doses of Spinraza which is administered via a lumber puncture.

After receiving the fourth, Heidi, who will also be starting physiotherapy, will then receive a 'maintenance' dose every four months.

Katie, who also has six-year-old daughter Delilah, said they had been told to expect to see improvements after the fifth dose but were trying not to get their hopes up too much.

"I'm looking forward to seeing how it helps her and what improvements she makes over the next six months.

"It's good to think that she won't get any worse now. If there no sign of improvements and this is as good at it gets then at least we know we can manage. Any improvements are a bonus," says Katie.