Medcare Women & Children Hospital successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to a three-year-old patient from Turkey, thus becoming one of the first private healthcare providers worldwide to offer this innovative treatment to an international patient, overcoming previous age and weight limitations.
SMA is a rare neuromuscular disorder that progressively weakens muscles responsible for movement and breathing. This newly licensed one-time therapy, which was first used in Abu Dhabi by Sheikh Khalifa Medical City (SKMC), part of SEHA and a subsidiary of PureHealth, under the supervision of the Department of Health – Abu Dhabi (DoH) two days ago, is developed by Novartis and approved by the US Food and Drug Administration (FDA) and UAE authorities last month.
For the three-year-old Hulus, daily routine earlier depended on intensive physiotherapy and hydrotherapy. However, the new procedure offers a promising alternative to lifelong injections. Hulus’ father, Mustafa, said the new therapy has renewed hope for greater independence and quality of life for his son.
Medcare pioneers SMA gene therapy
Dr Shanila Laiju, Group CEO of Medcare Hospitals & Medical Centres, said: “This is a defining moment for SMA care in the region. Families whose children were previously ineligible for gene therapy now have access to a revolutionary treatment that can significantly improve outcomes.
“This reflects Medcare’s long-standing commitment to children with rare diseases, supported by specialised expertise and advanced infrastructure.”
Since 2020, Medcare has treated more than 180 children with SMA from countries like Iran, Turkey, Nepal, Romania, Russia, Kazakhstan, Kyrgyzstan, Ukraine, Belarus, Lebanon, India, and Ethiopia, and reinforced Dubai’s role as a strategic global hub for medical tourism and paediatric genetic care.
Medcare delivers SMA care through a multidisciplinary model encompassing neurology, pulmonology, orthopaedics, rehabilitation, genetic counselling, and long-term follow-up for international patients.
Dr Vivek Mundada, Consultant Paediatric Neurologist, added: “Older and heavier patients can now benefit from this transformative therapy. This shifts SMA care beyond infant survival toward preserving mobility, independence, and respiratory function in older patients.”
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