Abu Dhabi first in world to deliver ITVISMA gene therapy for spinal muscular atrophy
Abu Dhabi has achieved a global milestone by becoming the first in the world to administer ITVISMA (onasemnogene abeparvovec), a breakthrough gene replacement therapy for spinal muscular atrophy (SMA), Qazinform News Agency learnt from WAM.
The treatment was successfully delivered at Sheikh Khalifa Medical City (SKMC) under the supervision of the Department of Health – Abu Dhabi (DoH). Developed by Novartis, ITVISMA is a one-time therapy specifically designed to target the underlying genetic cause of SMA in patients aged 2 years and older with a confirmed SMN1 gene mutation.
The therapy received accelerated approval in the UAE on November 25, 2025, making the country one of the first globally — after the United States — to endorse this pioneering treatment.
Officials highlighted the achievement as a testament to Abu Dhabi’s role as a leading global healthcare destination.
Dr. Noura Khamis Al Ghaithi, Under-Secretary of DoH, said the milestone reflects Abu Dhabi’s commitment to genomics, precision medicine, and access to cutting-edge therapies for rare diseases.
Bader Al Qubaisi, CEO of SKMC, emphasized the emirate’s integrated healthcare ecosystem and collaboration with Novartis.
Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, noted the impact on patients and families, underscoring Abu Dhabi’s growing role as a reference center for advanced neuromuscular care.
As 2025 draws to a close, a review of the global scientific landscape by Qazinform News Agency shows a series of advances pointing to earlier disease detection, more effective medical treatment, and tangible progress in environmental recovery. From gene-editing breakthroughs and new medicines to biodiversity gains and rapid epidemic response, the year demonstrated how sustained research, policy coordination, and global cooperation can translate into measurable results.
