The United Arab Emirates has become the second country in the world to approve Itvisma, a breakthrough gene therapy for the treatment of Spinal Muscular Atrophy (SMA), marking a significant milestone in advanced healthcare and rare disease management. SMA is a severe genetic disorder that affects motor neurons, leading to progressive muscle weakness and, in its most serious forms, early childhood mortality.
The approval reflects the UAE’s growing role as a regional hub for cutting-edge medical innovation. Gene therapies like Itvisma are designed to address the root genetic cause of SMA rather than merely managing symptoms. By delivering a functional copy of the faulty gene responsible for the disease, the therapy aims to halt or significantly slow disease progression with a single treatment.
Health authorities in the UAE emphasized that the decision followed rigorous clinical evaluation and safety reviews, aligning with international regulatory standards. The move is expected to improve access to life-saving treatment for SMA patients in the region, reducing the need for families to seek care abroad.
Experts say the approval also signals broader confidence in gene-based medicine, which is increasingly seen as the future of treatment for inherited disorders. While such therapies remain costly, early access can dramatically improve quality of life and long-term outcomes, particularly when administered at a young age.
The UAE’s decision underscores its commitment to innovation-driven healthcare, personalized medicine, and early adoption of transformative medical technologies. It also strengthens global momentum toward wider acceptance and regulation of gene therapies for rare and previously untreatable diseases.
