Dubai pioneers breakthrough gene therapy for rare diseases like SMA and DMD, transforming lives with one-time treatments led by global experts.
Dubai is fast emerging as a destination for advanced gene-based treatments, especially for children diagnosed with rare and life-threatening conditions like Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD). A key specialty facility, Medcare Royal Specialty Hospital in Al Qusais, Dubai is leading this medical transformation, offering groundbreaking therapies that replace lifelong spinal injections with a single, potentially curative dose.
Turkey and beyond, particularly as Turkey faces higher-than-average incidence rates of SMA—affecting 1 in every 6,000 births compared to 1 in 10,000 globally. Turkish health authorities estimate over 3,000 individuals living with SMA and around 150 new diagnoses each year. DMD, another progressive neuromuscular disorder, affects over 4,000 genetically confirmed patients in Turkey.
Medcare Royal Speciality Hospital’s specialized pediatric neurology unit, under the guidance of Dr. Vivek Mundada, British Board-certified Consultant Paediatric Neurologist, has achieved remarkable results. More than 100 gene therapy infusions for SMA have been successfully completed, making it the first private provider outside the US to do so in the MENA region.
Patients receive care through a holistic, multidisciplinary approach involving pulmonology, orthopaedics, rehabilitation, and genetic counselling. Post-treatment, international families are supported through long-term follow-ups to ensure continuity of care.
The impact of this approach is visible in cases like Mirha, diagnosed with SMA at just nine days old and treated in 2024. Today, she lives without the need for breathing or feeding support. Similarly, Elif Bade, who received therapy at age two, has shown significant developmental progress.
“We cannot express enough gratitude,” said Mirha’s parents. “Thanks to the expert team in Dubai, our daughter has a new future.”
A new rare disease unit at the hospital now features AI-powered diagnostics, robotic surgery, and advanced quaternary care, placing Dubai firmly on the global medical map. The facility operates under Aster DM Healthcare and aligns with the UAE’s ambition to be a global medical innovation hub.
For international patients, especially those from regions with limited access or long wait times, gene therapy in Dubai offers both a clinical breakthrough and a chance at a healthier life—today, not years from now.
