A private hospital in the United Arab Emirates has treated its first international patient with a newly licensed intrathecal gene therapy for spinal muscular atrophy, expanding access to advanced genetic treatment for older children and adults with the condition.
Medcare Women and Children Hospital in Dubai administered the one-time intrathecal gene therapy to a three-year-old child from Turkey, marking a milestone for spinal muscular atrophy care in the region. The treatment is designed to address the underlying genetic cause of the disease by delivering a functional copy of the SMN1 gene directly into the central nervous system.
While recent advances have transformed outcomes for infants diagnosed early, access to gene therapies has previously been limited by age and weight restrictions. The availability of this intrathecal approach allows treatment of older and heavier patients who were previously ineligible for gene replacement therapies.
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According to Medcare, the treated child had relied on intensive physiotherapy and hydrotherapy prior to the procedure. The gene therapy offers an alternative to ongoing, lifelong injectable treatments, with the potential to improve motor function, independence and quality of life. Hospital leaders described the case as a significant step forward for families affected by spinal muscular atrophy in the Middle East and beyond.
Clinicians involved in the programme highlighted that the therapy represents a shift in treatment goals, from focusing solely on infant survival to preserving mobility, respiratory function and long-term independence in older patients. Medcare delivers spinal muscular atrophy care through a multidisciplinary model, involving paediatric neurology, respiratory medicine, orthopaedics, rehabilitation, genetic counselling and long-term follow-up.
Since 2020, Medcare has reportedly treated more than 180 children with spinal muscular atrophy, including patients from Europe, Asia, the Middle East and Africa. The hospital’s role in treating international patients reflects Dubai’s growing position as a centre for medical tourism and specialised paediatric genetic care. Medcare, part of Aster DM Healthcare, has stated that it continues to invest in advanced technologies and specialist expertise to support care for rare and complex neurological conditions.
This news item has been summarised using AI and checked by humans before publication.
Sources
AETOSWire
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