Abu Dhabi Hospital Administers World’s First ITVISMA Gene Therapy for SMA

Abu Dhabi’s Sheikh Khalifa Medical City has become the first hospital in the world to administer ITVISMA, a one-time gene therapy for spinal muscular atrophy (SMA). The treatment was delivered on December 28, 2025, under the supervision of the Department of Health, Abu Dhabi.

Regulatory Approval and Global Significance
The UAE approved ITVISMA on November 25, 2025, making it the second country globally, after the United States, to authorise the therapy. The successful administration marks a major advance in precision and genomic medicine.

About ITVISMA
Developed by Novartis, ITVISMA directly targets the genetic cause of SMA by replacing the defective SMN1 gene, enabling the body to produce the survival motor neuron (SMN) protein, which is essential for motor neuron function.

Key Features of the Therapy

• Single-dose, one-time gene therapy
• Approved for patients aged two years and above, including adults
• Expands treatment access beyond infant-only SMA therapies
• Addresses the disease at its genetic root, unlike symptomatic treatments

About Spinal Muscular Atrophy (SMA)
SMA is a rare, progressive neuromuscular disorder that causes muscle weakness and degeneration. If untreated, it can lead to severe disability and life-threatening complications, particularly affecting breathing and mobility.