Following a long-running campaign to access an expensive new muscular dystrophy treatment, one very lucky six-year-old boy has begun trials of innovative new drug, Translarna, this week.
Luca Fernandes, Dorset, is one of 50 muscular dystrophy patients in England who has been given the rare chance to trial the experimental drug.
Translarna, which targets the legs to combat the muscle-wasting effects of muscular dystrophy, was first unveiled in April 2016.
Luca’s parents have been campaigning for the six-year-old to be given the £200,000 treatment for more than two years.
If untreated, Luca’s condition could leave him unable to walk by the age of 12, should his current condition deteriorate.
According to a number of medical professionals, the Translarna drug, which the Fernandes family has described as being like sachets of “powdered hope,” should be able to delay the damage caused by Duchenne muscular dystrophy for up to seven years.
“Our hope is, it’s going to give him more time on his feet, more time out of a wheelchair,” said mother, Joanne Fernandes.
“It’s going to allow him to keep playing football, go out in the playground with his friends and lead a normal life for much longer.”
“We know he is getting the best that he can get – there is nothing more we can do. We’ve fought so hard for this drug,” she added.
EnViva Paediatric Care, our experienced staff can provide complex nurse-managed homecare for babies, children and young people with a range of disabling conditions including muscular dystrophy. For more information about the services we provide, please contact us today.