Abu Dhabi’s Sheikh Khalifa Medical City has become the first hospital globally to administer ITVISMA, a one-time gene therapy for spinal muscular atrophy, marking a milestone in precision medicine. The treatment was delivered on December 28, 2025, under the supervision of the Department of Health, Abu Dhabi, following the UAE’s regulatory approval on November 25, making the country the second in the world after the United States to authorise the therapy.
Developed by Novartis, ITVISMA targets the genetic root of spinal muscular atrophy by replacing the defective SMN1 gene, enabling the production of the survival motor neuron protein. Unlike existing options, it is a single-dose therapy approved for patients aged two years and above, including adults, widening access beyond infant-only treatments.
Spinal muscular atrophy is a rare and progressive neuromuscular disorder that leads to muscle weakness and can cause life-threatening complications. The new therapy offers the potential for sustained improvement in motor function and reduced dependence on long-term treatment. Dr Noura Khamis Al Ghaithi, under-secretary of the Department of Health, Abu Dhabi, said, “This milestone reflects Abu Dhabi’s commitment to delivering world-class care and strengthening its position as a global leader in genomics and precision medicine.”
While the exact cost of ITVISMA has not been disclosed, it is expected to be substantial, similar to Zolgensma, which has been priced around $2 million per dose, underscoring ongoing discussions about accessibility for rare disease therapies.
