An eight-year-old boy living with muscular dystrophy could get access to a pioneering new treatment within a matter of weeks after a new deal was agreed by health bosses.
The National Institute for Clinical Excellence (NICE) is expected to publish guidance on the use of a drug which it is hoped will slow the progress of the muscle-wasting condition that Jagger Curtis was born with.
For many months the Hampshire family have been lobbying the medication watchdog to approve access to Translarna, which has been shown to slow down symptoms in certain circumstances.
Jagger’s dad James said it was hugely important that the drug be made available as quickly as possible.
“They said it could be autumn before we get it but we think he needs it now,” he said.
“Hopefully it won’t be too long. He’s getting a little bit more achy. But he’s happy and positive and that’s the main thing.”
NICE recommended that Translarna be made available on the NHS back in April, following a lengthy lobbying campaign by those families devastated by the life-limiting condition.
Robert Meadowcroft, Muscular Dystrophy UK’s chief executive, said: “While NICE and NHS England have been conducting their assessment of Translarna, families have faced a desperately long and frustrating wait, and had to endure the heartbreak of seeing their sons decline, while this licensed drug was not available to them.
“We give the boys and their families our huge thanks and recognition of their persistent and dedicated campaigning efforts and, after working together with many charities, we are pleased we are now a significant step closer to Translarna at last being made available to eligible boys on the NHS in England.”
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.