The UAE has approved the use of a gene therapy to advance the treatment of adults and children with spinal muscular atrophy.
Itvisma, developed by Swiss pharmaceutical company Novartis, works to replace the defective SMN1 gene responsible for the debilitating disease. Delivered in a one-time fixed dose, it aims to improve quality of life and reduce reliance on long-term treatments.
The Emirates Drug Establishment granted regulatory approval following clinical trials that demonstrated sustained improvements in patients' motor abilities.
The UAE is only the second country to authorise Itvisma's use – for adults and children aged two and above – after it was approved by the US Food and Drug Administration in November.
What is spinal muscular atrophy?
SMA affects motor neurons, which are nerve cells that send impulses to the muscles, and causes limb weakness.
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