A four-year-old boy from East Lothian has become the first child in the UK with a rare and deadly muscle-wasting disease to receive NHS funding for a breakthrough therapy.
Cormac Fegan will begin receiving the drug Translarna after he reaches his fifth birthday in November for the condition Duchenne muscular dystrophy.
The treatment is the first available in the EU for the underlying cause of the life-limiting muscle-wasting condition, and could keep Cormac walking for longer.
Health professionals confirmed to the boy’s parents they would gain access to the drug following the approval of an Individual Funding Request by Lothian Health Board.
Translarna has been available in several European countries since it was approved by the EU in August last year.
Gary Fegan, Cormac’s father, said: “This is really exciting for us - we feel very lucky that the timings have worked out, as Cormac is about to have his fifth birthday, the age when you can start taking Translarna.
"This gives us hope for the future and when new and innovative drugs come through in Scotland, Cormac should be eligible.
"We felt a huge sense of relief when we received the news."
Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said: "This is a true landmark moment.
"It is the first time a therapy addressing a genetic cause of Duchenne muscular dystrophy will be funded by the NHS.
"We hope that Cormac’s case will be used as a benchmark for all the families in Scotland, and indeed the rest of the UK."
He added: "Duchenne is a life-limiting condition, and we must protect children and their quality of life.
"The opportunity to be able to walk for longer can be immeasurably precious.
"We may not yet be able to halt the difficult challenges these children face, but with this drug, we have the chance to delay them."
Duchenne muscular dystrophy causes muscles to weaken and waste, causing increasingly severe disability.
Most of the 100 children born in the UK with the condition each year are full-time wheelchair-users by the age of 12, and are at risk from life threatening heart and breathing problems from their teenage years. Few currently live to see the age of 30.
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