Toddler given new hope by $2 million donation for pioneering care in UAE

The Turkish parents of a young boy diagnosed with a life-threatening genetic disorder have hailed the hospital that offered him a second chance in life, thanks to donors who raised $2 million to pay for his care.

In November regulators in the UAE fast-tracked a novel gene therapy to treat the debilitating symptoms of spinal muscle atrophy (SMA). The severe genetic condition is characterised by degeneration of motor neurons, resulting in progressive muscle weakness, impaired breathing, and loss of motor function.

Successful trials of the therapy demonstrated sustained improvements in patients' motor abilities, prompting UAE regulators to approve the single-dose drug Itvisma, made by the Swiss pharmaceutical company Novartis.

One of the first to benefit was Turkish toddler Hulusi Sert, four, who was administered the treatment last month in Dubai, after his parents Mustafa, 32, and Remziye Sert, 30, raised $2 million from community donors in Turkey. It took the family 29 months to raise the money.

Hulusi Sert with his family. Antonie Robertson / The National

“When Hulusi was three or four months old, he started getting shaking hands and legs, so we knew something was wrong with him,” Mr Sert, who works in a hospital in Tekirdag delivering food for recovering patients, told The National. “We took him to see the doctors, but they were saying there was nothing wrong and that it was just a psychological issue.

“We finally approached one neurologist who directly asked if the hospital had done a test for SMA, and they said no because no one had asked for one. They did the test and when they got the result, he was diagnosed with SMA. He was 14 months old then.”

Transformative results

Gene-based therapies have transformed outcomes for affected children, particularly when delivered early and within specialised, multidisciplinary care environments.

The biggest challenge is paying for the treatments, which are among the most expensive in the world. Typically only the highest insurance plans cover the costs, leaving families to find other sources of fundraising or charity donors.

“It was really difficult to fund this in Turkey, as it was such a large amount,” Ms Sert told The National. “Because it was taking so long, Hulusi started losing movement in his legs. He stopped walking, and couldn’t use his legs so we had to get him into a wheelchair.

“It was very difficult for us, but thankfully through community donations we were able to raise enough money for this treatment in Dubai. We needed authorisation from the government to start fundraising, otherwise there was nothing from their side other than to transfer the funds directly to the hospital.

“From the beginning, we didn't know anything about SMA, or understand anything about how severe it is. After having a child diagnosed with this disease, we started learning each part of this disease pretty quickly.”

Hulusi is likely to remain in ho