Abu Dhabi’s Sheikh Khalifa Medical City has set a global benchmark in advanced healthcare by becoming the first hospital worldwide to administer ITVISMA, a one-time gene therapy for spinal muscular atrophy (SMA).
The treatment was successfully delivered on December 28, 2025, under the oversight of the Department of Health – Abu Dhabi, following regulatory approval granted on November 25. With this decision, the UAE became only the second country after the United States to authorise the therapy, reinforcing its growing leadership in precision medicine.
Developed by Novartis, ITVISMA addresses SMA at its genetic source by replacing the faulty SMN1 gene. This enables the body to produce the survival motor neuron protein essential for muscle function. Unlike existing therapies that often require repeated doses or are limited to infants, ITVISMA is a single-dose treatment approved for patients aged two years and above, including adults, significantly expanding access.
Spinal muscular atrophy is a rare, inherited neuromuscular condition that causes progressive muscle weakness and can lead to severe, sometimes life-threatening complications. By targeting the root cause of the disease, the new therapy offers the possibility of long-term improvement in motor function while reducing the need for ongoing treatment.
Dr Noura Khamis Al Ghaithi, Under-Secretary of the Department of Health, Abu Dhabi, described the development as a defining moment for the emirate’s healthcare ecosystem. She said the milestone reflects Abu Dhabi’s commitment to delivering world-class medical care and strengthening its position as a global hub for genomics and precision medicine.
While the official price of ITVISMA has not been disclosed, it is expected to be high, comparable to other breakthrough gene therapies such as Zolgensma, which has been priced at around $2 million per dose. The cost highlights the broader global debate around affordability and access to life-changing treatments for rare diseases.
With this achievement, Abu Dhabi continues to push the boundaries of medical innovation, translating regulatory ambition and scientific progress into tangible outcomes for patients worldwide.
