Sheikh Khalifa Medical City in Abu Dhabi became the first hospital in the world to give ITVISMA, a one-time gene replacement therapy for spinal muscular atrophy. The landmark treatment was administered on December 28, 2025, under the supervision of the Department of Health in Abu Dhabi. With this decision, the UAE became only the second country, after the US, to approve the therapy for clinical use.
ITVISMA, developed by Novartis, directly tackles the genetic cause of spinal muscular atrophy by replacing the defective SMN1 gene, which is important for the production of the survival motor neuron protein. Unlike other treatments that require regular doses throughout a lifetime, ITVISMA is intended to be administered once and has the potential to be used for lengthy periods of time. Importantly, the medication is licensed for patients aged two and above, expanding access beyond infants and young children.
Spinal muscular atrophy is a rare, inherited neuromuscular disorder that causes progressive muscle weakness and can result in serious respiratory and mobility complications. Health officials believe the therapy has the potential to considerably improve motor outcomes while reducing long-term treatment dependency. The medication is expected to be costly, raising concerns over affordability and access.
