Abu Dhabi Makes History With New Spinal Muscular Atrophy Treatment

Abu Dhabi has reached a historic milestone in spinal muscular atrophy treatment by becoming the first globally to successfully administer ITVISMA (onasemnogene abeparvovec) gene therapy. This medical breakthrough represents important progress in spinal muscular atrophy (SMA) research and brings new hope to patients with this genetic condition. The innovative therapy was administered at Sheik Khalifa Medical City under the Department of Health – Abu Dhabi’s supervision.

The UAE’s healthcare system achieved another milestone when this revolutionary SMA medicine received fast-track approval on November 25, 2025. This makes the UAE one of the first countries after the United States to approve this innovative treatment. ITVISMA works as a one-time gene therapy that targets why spinal muscular atrophy happens in patients aged two years and older who have a confirmed SMN1 gene mutation. The treatment’s effectiveness is backed by clinical data showing substantial improvements in motor function that lasted throughout the 52-week follow-up period.

Abu Dhabi administers world’s first ITVISMA gene therapy

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Medical teams at Sheik Khalifa Medical City successfully administered the breakthrough ITVISMA gene therapy under strict medical protocols. Quick coordination between regulatory authorities and hospital administrators made this medical milestone possible, ensuring the patient got treatment on time.

ITVISMA shows most important progress in spinal muscular atrophy medicine. The therapy targets the condition’s genetic root cause instead of just managing symptoms. Motor neuron cells receive a working copy of the human SMN1 gene that replaces the faulty gene causing disease progression.

Clinical trials before this treatment showed that patients gained meaningful improvements in muscle strength and motor function. On top of that, it improved their breathing and reduced their need for breathing support.

Abu Dhabi’s Department of Health worked with international medical experts throughout the procedure. They created protocols to monitor patients and provide follow-up care. This collaborative effort reinforces Abu Dhabi’s position as a leading center for advanced spinal muscular atrophy research and treatment.

The medical team delivered the gene therapy through an IV infusion in about an hour. They monitored the patient carefully to check for any negative reactions. The patient’s progress and the therapy’s effectiveness will be tracked through ongoing follow-up care.

UAE accelerates approval and delivery of spinal muscular atrophy treatment

The UAE became the second country after the United States to approve ITVISMA when the Emirates Drug Establishment (EDE) fast-tracked its authorization on November 25, 2025. This quick regulatory decision shows UAE’s dedication to getting innovative medical treatments to patients faster.

Dr. Fatima Al Kaabi, Director General of EDE, stated that the approval “demonstrates the UAE’s commitment to providing patients with the latest advanced genetic therapies, particularly those with rare genetic diseases like spinal muscular atrophy”. Detailed clinical evidence backed this decision, showing lasting improvements in patient’s motor abilities and consistent safety results.

Abu Dhabi’s all-encompassing approach to handling rare genetic disorders includes universal screening, quick confirmatory testing, and specialist referrals. The Department of Health plays a vital part by conducting rapid health technology assessments and speeding up product coding to make innovative treatments available quickly.

Mohamed Ezz Eldin, Head of the GCC Cluster at Novartis, highlighted how EDE works closely with healthcare partners to provide equal treatment access, especially when you have patients who couldn’t get one-time gene therapy before. This regulatory achievement strengthens UAE’s position as a regional hub for pharmaceutical breakthroughs and builds international trust in its regulatory system.

Officials highlight Abu Dhabi’s leadership in genomic medicine

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Abu Dhabi health officials take pride in the emirate’s pioneering role in genomic medicine after the historic ITVISMA gene therapy administration. Dr. Ahmed Al Mansouri, Director of Advanced Therapeutics at the Department of Health – Abu Dhabi, believes this achievement makes the emirate a global leader in spinal muscular atrophy treatment.

“This milestone illustrates our commitment to advancing genomic medicine and offering cutting-edge solutions for patients with rare genetic conditions,” stated Dr. Al Mansouri. Abu Dhabi’s complete genomic capabilities have grown through investments in research infrastructure and specialist training programs.

The successful ITVISMA therapy is part of Abu Dhabi’s Genome Program that positions the emirate as a pioneer in tailored medicine. The emirate’s growing network of specialized facilities focuses on spinal muscular atrophy research and treatment options.

Dr. Samira Al Hashimi, Chief Medical Officer at Sheik Khalifa Medical City, sees this therapy administration as proof of Abu Dhabi’s healthcare system’s growth. “Our ability to deliver such advanced spinal muscular atrophy medicine reflects years of capacity building and knowledge transfer,” she noted.

This achievement lines up with Abu Dhabi’s healthcare vision to become a regional hub for treating complex genetic disorders and developing innovative approaches to previously untreatable conditions.

Abu Dhabi’s successful administration of ITVISMA gene therapy marks a breakthrough moment for spinal muscular atrophy patients worldwide. The UAE now leads genetic medicine through this groundbreaking achievement that shows its steadfast dedication to developing healthcare solutions for rare genetic disorders. The therapy’s power to target the root cause instead of just treating symptoms has created a fundamental change in SMA treatment approaches.

Quick regulatory approvals explain the Emirates’ commitment to making innovative treatments available without delays. Sheik Khalifa Medical City has grown into a center of excellence through this innovative procedure. The Department of Health – Abu Dhabi continues to deepen their commitment to the emirate’s status as a regional hub for advanced medical therapies.

Clinical evidence for ITVISMA reveals promising results as patients show sustained motor function improvements. Patients who had limited treatment options can now access potentially life-changing therapy. A detailed approach from universal screening to specialized care has created a model healthcare system that manages complex genetic conditions effectively.

This early medical milestone paves the way for future gene therapy applications. The UAE’s investment in genomic medicine infrastructure has definitely yielded returns by creating an ecosystem where innovative treatments flourish. Healthcare professionals will learn about long-term outcomes through ongoing monitoring, which could benefit SMA patients globally.

This achievement proves Abu Dhabi’s vision of healthcare excellence and innovation. Careful planning, international collaboration, and steadfast dedication to patient care have turned this vision into reality. SMA treatment has a bright future as Abu Dhabi revolutionizes genetic medicine from theory into clinical practice.