Khaberni - Abu Dhabi has achieved a pioneering milestone, becoming the first in the world to offer gene therapy "Zolgensma", "Onasemnogene Abeparvovec" for spinal muscular atrophy, which enhances the emirate's position among the top health destinations and exemplifies its approach to patient care through the use of genomics and precision medicine.
Under the supervision of the Department of Health–Abu Dhabi, this innovative treatment was provided at the Sheikh Khalifa Medical City, and " Zolgensma" is a gene therapy developed by "Novartis", given once and specifically designed to target the fundamental genetic cause of spinal muscular atrophy for patients aged two years and older, who have a confirmed mutation in the "SMN1" gene.
The treatment received the expedited approval pathway in the UAE on November 25, making it one of the first countries in the world after the United States to adopt this revolutionary treatment. This achievement reflects Abu Dhabi's firm commitment to providing patients in the region with the latest therapeutic innovations.
"Zolgensma" was designed to be long-lasting, functioning to replace the lost "survival motor neuron protein 1" gene, which contributes to the improvement of motor functions and reduces the need for ongoing and repeated treatments that other therapeutic options require.
Innovative Treatment
In this regard, Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health–Abu Dhabi said: "This achievement reflects Abu Dhabi's approach in providing health care of global quality standards, establishing its leading status in health care based on genomics and precision medicine. We take pride in being among the first in the world to offer the innovative treatment " Zolgensma", which reaffirms Abu Dhabi's leading and accelerating role in innovation within the advanced health care sector. Our priorities focus on ensuring the health and well-being of community members in the emirate and beyond, facilitating access to advanced treatment options for rare diseases, enhancing the emirate's status as a leading destination for medical tourism, and this achievement enriches our distinguished record and underscores our commitment to providing the latest therapeutic solutions and health innovations that enhance the quality of life of patients".
