Dubai hospital treats boy, 3, with pioneering spinal muscular atrophy drug in 'defining moment'

Young patient from Turkey receives Itvisma gene therapy at Medcare Women and Children Hospital

Medcare Women and Children Hospital has administered Itvisma to three-year-old Hulus, from Turkey. Photo: AETOSWire

A Dubai hospital has delivered a pioneering gene therapy treatment to a three-year-old boy with spinal muscular atrophy, in what has been hailed as a “defining moment” for medical care in the region.

Doctors at Medcare Women and Children Hospital administered the single-dose drug Itvisma to Hulus, an international patient from Turkey.

The drug, developed by Swiss pharmaceutical company Novartis, received accelerated approval by the UAE on November 25 after clinical trials demonstrated sustained improvements in patients' motor abilities.

SMA is a rare neuromuscular disorder that progressively weakens muscles responsible for movement and breathing. It can cause severe disability and, in some cases, proves fatal in young children.

Itvisma is a new version of Novartis' existing treatment, Zolgensma, which was only available to children under the age of two.

Itvisma can be administered to children over two years old or who weigh more than 21kg, offering a lifeline to patients such as Hulus, who otherwise faced years of painstaking treatments and regular injections.

“This is a defining moment for SMA care in the region,” said Dr Shanila Laiju, group chief executive of Medcare Hospitals and Medical Centres.

“Families whose children were previously ineligible for gene therapy now have access to a revolutionary treatment that can significantly improve outcomes.

“This reflects Medcare’s long-standing commitment to children with rare diseases, supported by specialised expertise and advanced infrastructure.”

The price of the drug has not been disclosed, but it works in a similar way to Zolgensma, which costs around $2 million per patient.

“Older and heavier patients can now benefit from transformative therapy,” said Dr Vivek Mundada, consultant paediatric neurologist at Medcare.

“This shifts SMA care beyond infant survival toward preserving mobility, independence and respiratory function in older patients.”

Since 2020, Medcare, a private healthcare provider that is part of Aster DM Healthcare, has used gene therapy to treat 180 children with SMA, including some who have travelled from Iran, Turkey, Nepal, Romania, India and Ethiopia.

Expanding support

On Sunday, Abu Dhabi's Sheikh Khalifa Medical City said it had become the first in the world to administer Itvisma. Authorities did not reveal details of the patient who received the treatment or when it took place.

Last week, Medcare Royal Specialty Hospital in Dubai's Al Qusais announced it was also poised to become one of the first centres outside the US to offer the SMA drug.