A young Syrian girl with spinal muscular atrophy received Dh7 million gene therapy paid for by Sheikh Mohammed bin Rashid, Vice President and Ruler of Dubai, on Friday at Dubai Health's Al Jalila Children’s Hospital.
Holding a small giraffe toy, and trying to smile, Yaqeen Kankar, sat in the treatment room on Friday morning, waiting for the arrival of doctors and her medicine. Two weeks ago, the family made a video appeal on social media for help to save the two-year-old.
The office of Sheikh Mohammed called the family to say Sheikh Mohammed would cover the cost of the treatment.
Yaqeen's uncle, Ibrahim Abdulaziz Faroj, told The National the family was relieved to know she could now have a normal life.
“We didn’t sleep for three days after receiving the happy news that Sheikh Mohammed would cover the cost,” Mr Faroj, 23, said. “It’s a mix of feelings between joy, anticipation and anxiety. We want her to grow normally like any other child. Thanks to Dubai, the dream becomes true.”
He had posted a video holding Yaqeen, explaining her condition and asking for help to pay for the treatment, which is available at only a few centres in the world.
“We know that she has no time. We didn’t have other way than to plea for help as we couldn’t afford the cost,” Mr Faroj said.
Eight months before moving to Dubai, doctors in Syria treating Yaqeen diagnosed spinal muscular atrophy, a genetic disorder that weakens and wastes muscles.
“I was posting about her for several months until we received the happy call,” Mr Faroj said. “It was the best gift ever. We all cried with joy when we heard the news. This action is not strange for the UAE and its rulers, who always help people in desperate need.”
Doctors gave Yaqeen only months to live without the treatment.
Dr Haitham Elbashir, paediatric neurorehabilitation consultant at Al Jalila Children’s Hospital, told The National: “We examined her a week ago and did all the laboratory tests. I'm very happy to say that the medicine is going to be delivered to her today.”
Yaqeen underwent the one-hour procedure on Friday and will stay in the hospital for 24 hours. She can then return with her parents to their home in Sharjah.
Doctors said they were optimistic it would be successful, but it could take up to three months to see the final results.
“She will need to continue with us for three months because that's the treatment period where we need to follow her regularly,” said Dr Elbashir.
“Her condition is stable, but she's got some weakness and hopefully the treatment will help to get her better. I’m very optimistic."
SMA affects the nerve cells that transmit impulses to the muscles and weakens the limbs. It makes walking difficult or impossible and creates problems with swallowing, as well as breathing.
The treatment offered by Al Jalila Children's Hospital, called Zolgensma, is given once and is a form of gene therapy. It was approved by the US Food and Drug Administration four years ago and has been described as the most expensive drug in the world.
The hospital has completed more than 100 treatments for patients with SMA, said Dr Mohamed Al Awadhi, executive director of the Dubai Health Women and Children’s Campus.
