Abu Dhabi first globally to deliver ITVISMA gene therapy for spinal muscular atrophy

Key facts

• ITVISMA (onasemnogene abeparvovec) is a one-time gene therapy targeting the underlying genetic cause of spinal muscular atrophy in patients aged 2 years and older with a confirmed SMN1 gene mutation.
• The therapy replaces the missing SMN1 gene to improve motor function and reduce the need for continuous treatments required by other therapies.
• Administered at Sheikh Khalifa Medical City (SKMC), part of SEHA, a subsidiary of PureHealth, under the supervision of the Department of Health – Abu Dhabi (DoH).
• Developed by Novartis and received accelerated UAE approval on 25th November 2025, positioning the UAE among the first countries globally after the USA to endorse this treatment.