Abu Dhabi Sets New Benchmark with ITVISMA Gene Therapy for SMA

Abu Dhabi has made headlines by becoming the world's first city to implement ITVISMA (onasemnogene abeparvovec) gene therapy for spinal muscular atrophy (SMA). This significant advance underscores the emirate's strength in the fields of genomics and cutting-edge medical solutions.

Supervised by the Department of Health – Abu Dhabi (DoH), the SEHA Sheikh Khalifa Medical City (SKMC), a part of SEHA and a subsidiary of PureHealth, has successfully administered this therapy created by Novartis. ITVISMA is a one-off treatment targeting the fundamental genetic issue causing SMA in patients aged two years and older with an identified SMN1 gene mutation.

On November 25, 2025, the UAE granted swift approval for ITVISMA, making it one of the first countries globally, following the US, to endorse this innovative treatment. Unlike typical therapies that necessitate ongoing administration, ITVISMA replaces the absent SMN1 gene, enhancing motor functions and offering prolonged benefits for patients.

Key Statements:

• Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, remarked:
  "This landmark event signifies Abu Dhabi’s dedication to top-notch care and innovative healthcare solutions. Administering ITVISMA solidifies our status as a global authority in genomics and precision medicine, ensuring patients access crucial therapies for rare conditions."

• Bader Al Qubaisi, CEO of SKMC, said:
  "Providing the world's first ITVISMA treatment exemplifies Abu Dhabi’s comprehensive healthcare framework. Our collaboration with partners like Novartis allows us to deliver safe and effective access to this game-changing therapy."

• Mohamed Ezz Eldin, Head of GCC Cluster at Novartis, stated:
  "This achievement is fundamentally about supporting patients and their families. Our partnership with DoH and SKMC is all about broadening access to pioneering treatments like ITVISMA, furthering Abu Dhabi’s role in advanced neuromuscular care."

Comprehensive SMA Care in Abu Dhabi:

Abu Dhabi’s strategy for addressing severe genetic disorders, including SMA, includes:

• Universal newborn screening along with whole genome sequencing for over 815 manageable genetic conditions.

• Prompt confirmatory testing and swift referrals to DoH-approved specialists and centers of excellence.

• Fast-tracked access to early therapies through efficient health technology evaluations and approval processes.

This cohesive model sets Abu Dhabi as a global leader in rare disease management, fusing innovation, precision medicine, and patient-focused treatment approaches.