Callum Smith: Limavady boy to get life-changing drug for muscle disease

Callum Smith
Image caption Callum Smith suffers from muscle cramps and is on heart medication.

The mother of a Limavady boy, who has a rare form of muscular dystrophy, said she is "delighted" that he will now receive treatment.

Seven-year-old Callum Smith suffers from Duchenne, a genetic disease that gradually causes weakness and loss of muscle function.

He will be one of around 10 children in Northern Ireland to benefit from the drug Translarna.

His mother Laura said: "It's emotional, we still can't quite believe it."

Callum receives daily physiotherapy and heart medication but is unable to jump, or run, and relies on his wheelchair for long distances.

"We always said that until we had the prescription in our hands it won't seem real to us because we came so close in the past," said Laura.

"God willing it will go really fast now and in a matter of weeks he'll be able to receive his drug."

Patients with the condition lack normal dystrophin, a protein found in muscles, which helps to protect muscles from injury.

Taking the drug, particularly before the age of 10, can help prevent a life spent in a wheelchair.

The Smith family had been campaigning for many years to receive Translarna.

Laura believes the drug will transform her son's life.

"We know of other boys on the drug in Scotland and to be able to see a child that age who could never ride a bicycle before going up and down hills on a bicycle, you wouldn't have thought there was anything wrong with him.

"We have heard from the research in the trials that it could extend his walking average for about 7 years, which is immense.

"We've had a lot of support along the way from a lot of the politicians and we are just so, so grateful that it has been made available," she said.

There are 2,400 children in the UK living with muscular dystrophy, but only those whose condition is caused by a particular 'nonsense mutation' - around 200 children - are suitable to use Translarna.

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