An 11-year-old boy with a rare muscle-wasting disease has begun one of the world’s most expensive medical treatments in Abu Dhabi, after a nationwide fundraising drive in Turkey.
Yigit Elma has Duchenne muscular dystrophy, a rare inherited condition that almost exclusively affects boys and has no cure. Hope arrived for the youngster and his family when they learnt about Elevidys, a one-time gene therapy available in the UAE.
The family could not afford the $2.8 million treatment on their own. But Yigit’s plight united thousands of people across Turkey, as neighbours, businesses, sports clubs and strangers rallied to give the football-loving boy a chance of being treated.
He has now been admitted to Burjeel Medical City, where doctors are preparing him to receive Elevidys. He will be the first patient with Duchenne muscular dystrophy to undergo gene therapy at the hospital.
“Without treatment, the condition will continue to deteriorate,” Prof Ayman El-Hattab, director of the hospital's genetics and rare disease centre, told The National. “Gene therapy is not a cure, but it can slow the progression of the disease and improve muscle strength.”
Charity football matches, volleyball tournaments, community iftars, fund-raising appeals and the auction of a car helped raise the money for Yigit. The campaign also spread beyond Turkey through social media and community networks before bringing him to Abu Dhabi.
“Our community and our country stood beside us throughout this journey,” Ismail Elma, Yigit’s father, told The National. “People who had never even met us helped our son. We could never have reached this point without their support."
He hailed the medicine and expertise at Burjeel Medical City, which he said "give hope not only to Yigit, but to many other families facing the same condition". He expressed hope that his son would improve enough for him to "chase his dreams".
Duchenne muscular dystrophy is caused by mutations in the dystrophin gene. Children often begin life walking and playing normally before gradually finding it harder to run, climb stairs or stand up. As the disease progresses, many lose the ability to walk and later develop complications affecting their heart and breathing muscles.
Yigit's family first had cause for concern when he was five, with his parents noticing his legs were unusually swollen. They visited hospitals in Konya, Ankara and Istanbul in search of answers before genetic testing confirmed the disease.
“We couldn’t believe it,” Rabia Elma, Yigit’s mother, told The National. “We went to almost every major hospital in Turkey because we hoped someone would tell us the diagnosis was wrong. In the end, we had to accept it. It was one of the saddest moments of our lives.”
Like many families, they had never heard of the disease before Yigit’s diagnosis. Over the following years, his muscles gradually weakened, making everyday tasks increasingly difficult.
After one fall at school, his parents made the difficult decision to withdraw him and educate him at home, fearing another accident. But the previously outgoing boy became quieter and more reserved as he spent less time with classmates and friends.
For his family, the gene therapy represents far more than a medical milestone. They hope it will allow Yigit to reclaim some of the childhood that the disease has taken away.
Yigit, whose name means “brave” in Turkish, has a simple dream. “I want to play football with my friends again,” he said.
For his parents, it is a simple wish that means everything. For Yigit, it is the hope of one day returning to the football pitch, riding a bicycle and going back to school – the ordinary experiences that thousands of people across Turkey came together to help him get back to.
