A schoolboy living with a chronic muscle-wasting condition is facing uncertainty over whether he will get access to a newly-approved drug, even though he has been involved in trialling it.
It was confirmed just a few days ago that NICE, the UK’s medicines regulator, had approved Translarna for use to help relieve the symptoms of some children living with Duchenne Muscular Dystrophy.
Ten-year-old Leo Le Gal had started to test Translarna around two years ago and his parents say the medication has helped maintain his core strength; the youngster still has good mobility in his arms.
However, because Leo has lost the use of his legs and the drug has only been approved for those who can still walk, there are now doubts over whether he will have access to the treatment – despite the fact that it has helped to stabilise his condition.
Leo’s mother Ruth Le Gal, from Ivybridge, Devon, said it was “fantastic news” that other children would now be entitled to NHS funding for Translarna, but she said that uncertainty was hanging over her own family.
“It’s great for all those children that will be eligible but sadly Leo won’t because, unfortunately he lost the ability to walk before he was able to start taking the medicine,” she told BBC News.
“We have no idea what will happen after that,” she said, voicing hopes that the trial may yet be extended.
Leo was diagnosed with muscular dystrophy at the age of five after his mother, who had previously worked as a physiotherapist, noticed something was wrong.
At EnViva Paediatric Care, our experienced staff can provide complex nurse-managed homecare for babies, children and young people with conditions including muscular dystrophy. For more information about the services we provide, please contact us today.