Abu Dhabi Pioneers Global Gene Therapy for Spinal Muscular Atrophy with ITVISMA

Abu Dhabi has achieved a world-first by administering ITVISMA, a groundbreaking one-time gene replacement therapy for spinal muscular atrophy, under the supervision of the Department of Health - Abu Dhabi at Sheikh Khalifa Medical City. The therapy, developed by Novartis, targets the underlying SMN1 gene mutation in patients aged two and older, offering long-term improvement in motor function. This milestone reinforces Abu Dhabi's position as a leading global healthcare destination, with accelerated approval in the UAE positioning it among the first countries globally to endorse this treatment. The achievement highlights the emirate's commitment to genomics, precision medicine, and providing cutting-edge therapies for rare diseases to the community and medical tourists.

Key Points: Abu Dhabi First to Administer ITVISMA Gene Therapy for SMA

  • First global delivery of ITVISMA for SMA
  • Targets genetic cause in patients 2+
  • UAE among earliest adopters after USA
  • Reduces need for continuous treatment
3 min read

Abu Dhabi leads world in breakthrough gene replacement therapy 'ITVISMA' for spinal muscular atrophy

Abu Dhabi becomes the first globally to deliver ITVISMA, a breakthrough one-time gene replacement therapy for spinal muscular atrophy, marking a milestone in genomics and precision medicine.

"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. - Noura Khamis Al Ghaithi, DoH Under-Secretary"

Abu Dhabi, December 28

Abu Dhabi marks a landmark achievement in becoming the first in the world to deliver ITVISMA for the treatment of spinal muscular atrophy, reinforcing the emirate's position as a leading global healthcare destination and demonstrating its commitment to patient care through genomics and precision medicine.

Under the supervision of the Department of Health - Abu Dhabi (DoH), the regulator of the healthcare sector in the emirate, Sheikh Khalifa Medical City (SKMC), part of SEHA, a subsidiary of PureHealth, successfully administered this groundbreaking 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.

This groundbreaking therapy received accelerated approval in the UAE on November 25, 2025, positioning the UAE among the first countries globally, after the USA, to endorse this pioneering treatment. This underscores Abu Dhabi's commitment to bringing cutting-edge innovations to patients across the region.

Designed for simplicity and long-term impact, ITVISMA replaces the missing SMN1 gene to improve motor function, reducing the need for continuous treatments required by other therapies.

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 and accelerator in advanced and innovative healthcare. Our priority remains safeguarding the health of our community members and beyond, ensuring access to cutting-edge therapies for rare diseases, supporting the emirate's standing as a leading destination for medical tourism. This achievement is just one of many as we remain dedicated to accelerating access to life-changing therapies and providing patients with the latest advancements in healthcare."

Bader Al Qubaisi, Chief Executive Officer at SKMC, said, "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. In line with PureHealth's vision and DoH's guidance in defining seamless care pathways and facilitating collaboration with partners such as Novartis, we have been able to bring this life-changing therapy to our patients safely and efficiently."

Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, added, "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 such as ITVISMA, and to contribute to Abu Dhabi's growing role as a regional and global reference for advanced neuromuscular care."

- ANI

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Reader Comments

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Rohit P
Amazing achievement by Abu Dhabi. But it also makes me think - when will such gene therapies become accessible and affordable in India? Our healthcare system needs to accelerate in adopting these cutting-edge treatments. The cost is a major barrier for most Indian families.
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David E
Working in biotech, I know how complex this therapy is. For Abu Dhabi to be second only to the USA in administering it is a massive statement. The collaboration between the health department, hospital, and Novartis seems seamless. A model for other regions to follow.
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Ananya R
One-time gene replacement therapy that targets the root cause? This is the future of medicine. Hats off to the scientists and the vision of the Abu Dhabi leadership. Hope this success encourages more investment in genomic research in South Asia as well.
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Suresh O
While this is a positive step, I hope the focus on "medical tourism" doesn't mean this treatment remains out of reach for ordinary citizens in the region and neighboring countries. Healthcare breakthroughs should prioritize local need, not just global prestige. A respectful criticism.
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Kavya N
My cousin's child has SMA. The daily struggles are real. News like this is a beacon of light. The fact that it reduces the need for continuous treatment is a game-changer for quality of life. More power to the researchers and doctors involved! ❤️

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