Abu Dhabi Becomes First Worldwide to Deliver ITVISMA Gene Therapy for Spinal Muscular Atrophy

Abu Dhabi has achieved a global healthcare milestone by becoming the first in the world to administer ITVISMA (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) patients aged two years and older with a confirmed SMN1 gene mutation. The therapy, developed by Novartis, was delivered at Sheikh Khalifa Medical City under the supervision of the Department of Health – Abu Dhabi, highlighting the emirate’s leadership in genomics and precision medicine.

Pioneering One-Time Gene Therapy
ITVISMA is designed to replace the missing SMN1 gene, addressing the root cause of SMA and improving motor function with a single administration. The UAE granted accelerated approval for the therapy on 25 November 2025, making it one of the first countries globally, after the USA, to endorse this innovative treatment, which reduces reliance on continuous therapies and significantly enhances patient quality of life.

Abu Dhabi’s Growing Role in Advanced Healthcare
The administration of ITVISMA reflects Abu Dhabi’s commitment to positioning itself as a global healthcare hub and advancing medical innovation. Through integrated efforts by PureHealth, SKMC, and the Department of Health, the emirate continues to accelerate access to cutting-edge therapies, supporting rare disease patients while enhancing its reputation as a leading destination for advanced healthcare and medical tourism.