Abu Dhabi leads world in gene therapy to treat spinal muscular atrophy

Monday, 29 December 2025 07:13

By ARN News Staff

Abu Dhabi has become the first in the world to deliver Itvisma, a one-time gene therapy specifically designed to target the underlying genetic cause of spinal muscular atrophy (SMA) in patients aged two years and above.

Sheikh Khalifa Medical City (SKMC) has successfully administered this groundbreaking gene therapy, developed by Novartis, under the supervision of the Department of Health – Abu Dhabi (DoH), the regulator of the healthcare sector in the emirate.

The therapy had been previously approved by the UAE on November 25, positioning it among the first countries globally, after the USA, to endorse the pioneering treatment.

Designed for simplicity long-term impact, Itvisma (onasemnogene abeparvovec) replaces the missing SMN1 gene to improve motor function, reducing the need for continuous treatments required by other therapies.

Dr. Noura Khamis Al Ghaithi, Under-Secretary of DoH, stated that “this milestone reflects Abu Dhabi’s commitment to delivering world class care and strengthening its position as a global leader in healthcare driven by genomics and precision medicine."

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