Abu Dhabi has become the first place in the world to deliver ITVISMA, a breakthrough gene replacement therapy for spinal muscular atrophy (SMA), marking a major leap in advanced genetic medicine and rare disease care. Under the supervision of the Department of Health – Abu Dhabi, Sheikh Khalifa Medical City successfully administered the world’s first ITVISMA treatment, developed by Novartis. This cements Abu Dhabi’s position as a global hub for genomics and precision medicine.
ITVISMA received accelerated approval in the UAE on 25 November 2025, shortly after clearance in the United States, making the country among the first globally to authorise this therapy. Officials say this reflects a strong commitment to faster access to life‑changing treatments for rare disorders. ITVISMA is a one‑time gene therapy that targets the root genetic cause of SMA by replacing the faulty or missing SMN1 gene. It is approved for patients aged two years and older with a confirmed SMN1 mutation, including adults, which expands treatment options beyond early‑infant therapies.
By restoring production of the survival motor neuron protein, the therapy aims to improve motor function and reduce dependence on long‑term repeated treatments. Clinical studies cited at approval showed sustained benefits in motor abilities over 52 weeks, with a manageable safety profile. SMA is a rare, progressive neuromuscular disease that causes severe muscle weakness and can lead to life‑threatening complications. Access to a single‑dose gene therapy offers new hope to families who previously relied mainly on continuous drug regimens.
Health leaders in Abu Dhabi say this achievement demonstrates the strength of the emirate’s integrated healthcare ecosystem and its vision to become a reference centre for advanced neuromuscular care in the region and beyond.
FAQs [Frequently Asked Questions]
1. What is ITVISMA?
ITVISMA is a one‑time gene replacement therapy for spinal muscular atrophy that works by replacing the faulty SMN1 gene. It helps the body produce the protein needed for motor neurons, aiming to improve muscle strength.
2. Who can receive ITVISMA treatment?
ITVISMA is approved for children and adults aged two years and older with confirmed mutations in the SMN1 gene causing SMA. Eligibility is decided by specialist doctors after genetic testing and clinical evaluation.
3. Why is Abu Dhabi’s role important?
Abu Dhabi is the first in the world to deliver ITVISMA, showing its leadership in precision medicine and rare disease care. This step can attract global patients and drive further innovation in advanced therapies.