THE parents of a boy with a muscle-wasting condition fear funding for the first-ever drug to target the cause might be rejected by the NHS.
Helen Duell, 38, and Paul Duell, 42, an aircraft engineer, of White Hart Lane, Portchester, have spoken of their fears as their five-year-old son Harry has the condition.
At just eight weeks old he was diagnosed with Duchenne muscular dystrophy, which causes muscles to weaken and waste, including the heart and vital breathing muscles. Helen lost her brother to the same condition.
A meeting was held to review whether the drug Translarna should be funded on the NHS, which Mr and Mrs Duell believe could help their son to keep walking for longer.
A meeting between the National Institute for Health and Care Excellence, a pharmaceutical firm, medical experts and a patients’ group took place this week to see if the drug could be funded by the NHS.
But 50 families will have to wait until February to find out if it is possible.
Mrs Duell, a civil servant, said: ‘Harry’s condition is always at the forefront of our minds.
‘We have to try and live a normal life, knowing that the Duchenne is life-limiting. It is slowing him down physically, making it harder for him to keep up with his friends.
‘With the help of Translarna, Harry would be able to walk and run for longer, play football and have a better quality of life for longer. We would not have to watch helplessly as he loses muscle strength as Duchenne takes over.
‘The wait on news about funding has been excruciating.
‘It has been so frustrating knowing that there is a drug available that will help our son, but we can’t have it just yet.’
Nice said it would not recommend NHS access to Translarna without more justification of its cost and evidence of its impact.
The drug is available in other countries such as Germany, France, Spain and Italy.