Abu Dhabi Hospital First to Administer Pioneering Gene Therapy

Abu Dhabi has made medical history after Sheikh Khalifa Medical City (SKMC) became the first in the world to administer a groundbreaking gene therapy for spinal muscular atrophy (SMA), reinforcing the emirate’s role as a global leader in advanced healthcare. 

Under the supervision of the Department of Health – Abu Dhabi (DoH), SKMC successfully delivered ITVISMA (onasemnogene abeparvovec) — a one-time gene replacement therapy developed by Novartis that targets the underlying genetic cause of SMA. This treatment is designed for patients aged two years and older with a confirmed SMN1 gene mutation, offering the potential to improve motor function and reduce dependency on ongoing treatments. 

The therapy received accelerated approval in the UAE on November 25, 2025, placing the country among the first in the world — after the United States — to endorse this innovative treatment. The move underscores Abu Dhabi’s commitment to bringing cutting-edge medical innovations to patients across the region and strengthening its position as a destination for precision medicine and medical tourism. 

What ITVISMA Means for Patients

ITVISMA works by replacing the missing or defective SMN1 gene, which is responsible for the progressive weakening of motor neurons in SMA. By addressing the root cause of the disease, the therapy has the potential to significantly improve patients’ motor abilities and overall quality of life. 

SMA is a severe genetic condition that affects nerve cells controlling muscles, leading to muscle weakness and, in some forms, respiratory and swallowing difficulties. Without treatment, the most severe types can be fatal in early childhood. 

Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, highlighted that this milestone reflects the emirate’s dedication to world-class healthcare driven by genomics and precision medicine. She emphasized Abu Dhabi’s role as both a regional and global healthcare leader and reaffirmed its commitment to expanding access to life-changing therapies for rare diseases. 

Bader Al Qubaisi, CEO of SKMC, described delivering the world’s first ITVISMA treatment as a testament to Abu Dhabi’s integrated healthcare ecosystem and collaborative partnerships that have made this achievement possible. 

With this successful administration of ITVISMA, Abu Dhabi sets a new benchmark in the treatment of spinal muscular atrophy, offering hope to patients and families affected by the condition. Healthcare experts say this development could accelerate the availability of advanced therapies across the region and further establish the UAE as a hub for innovative medical care.