Sunderland mum speaks as muscular dystrophy sufferers set to benefit from drug

Translarna could keep children with the severe muscle-wasting condition walking for longer.

The National Institute for Clinical Excellence (NICE) has announced that it has secured a Managed Access Agreement on the drug.

In August 2014, Translarna became the first drug addressing a genetic cause of Duchenne muscular dystrophy to be approved in the EU.

It has been available to families in some European countries for over a year.

Three-year-old William Calvert, of Hylton Castle, was diagnosed with the condition last year, and his mum Caroline says that although Translarna will not be able to help with his illness specifically, the announcement is huge for other children.

“Translarna is to treat something called “nonsense mutations” which is something William doesn’t have,” said Caroline, 28.

“But we’ve signed campaigns for it as we know children who will benefit from it.

“There was a child on a Translarna trial who went from not being able to walk to riding his bike.”

Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “Parents of children eligible for Translarna have fought courageously for this outcome and to give their children the chance to keep walking for longer.”