Abu Dhabi Becomes First in the World to Deliver ITVISMA Gene Therapy for Spinal Muscular Atrophy

Abu Dhabi, The Gulf Observer: Abu Dhabi has achieved a global healthcare milestone by becoming the first in the world to administer ITVISMA (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA), further solidifying the emirate’s position as a leading destination for advanced healthcare and demonstrating its commitment to patient care through genomics and precision medicine.

Under the supervision of the Department of Health – Abu Dhabi (DoH), Sheikh Khalifa Medical City (SKMC), part of SEHA and a subsidiary of PureHealth, successfully delivered this pioneering gene therapy developed by Novartis.

ITVISMA is a one-time gene therapy specifically designed to target the underlying genetic cause of SMA in patients aged two years and older with a confirmed SMN1 gene mutation. The therapy received accelerated approval in the UAE on 25th November 2025, making the country one of the first globally, after the USA, to endorse this groundbreaking treatment.

The therapy offers a long-term solution by replacing the missing SMN1 gene to improve motor function, reducing the need for ongoing treatments associated with other therapies.

Dr. Noura Khamis Al Ghaithi, Under-Secretary of DoH, said, “This milestone reflects Abu Dhabi’s commitment to delivering world-class care and strengthening its position as a global leader in healthcare driven by genomics and precision medicine. By administering ITVISMA, we are proud to be among the first to provide this innovative treatment, further reinforcing our role as a leader in advanced healthcare. Our priority remains safeguarding the health of our community and ensuring access to cutting-edge therapies for rare diseases, supporting the emirate’s standing as a premier medical tourism destination. This achievement is one of many as we continue to accelerate access to life-changing therapies and provide patients with the latest healthcare advancements.”

Bader Al Qubaisi, Chief Executive Officer at SKMC, added, “Delivering the world’s first ITVISMA treatment at SKMC is a testament to Abu Dhabi’s integrated healthcare ecosystem under the leadership of the Department of Health – Abu Dhabi. Guided by DoH and PureHealth’s vision for seamless care pathways, our collaboration with partners such as Novartis has enabled us to bring this life-changing therapy to patients safely and efficiently.”

Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, said, “Today’s milestone is ultimately about patients and families. By working closely with the Department of Health – Abu Dhabi and SKMC, we are proud to support accelerated access to breakthrough therapies like ITVISMA and contribute to Abu Dhabi’s growing role as a regional and global reference for advanced neuromuscular care.”