A toddler from Oman has undergone a landmark medical procedure at Tawam Hospital in the UAE — a one-time gene replacement therapy for Spinal Muscular Atrophy (SMA), a rare and potentially fatal genetic condition that progressively robs children of muscle function.
The 18-month-old girl was diagnosed with SMA, a disorder caused by the absence or malfunction of the SMN1 gene, which is responsible for sustaining the motor neurons that control movement. As those neurons deteriorate, children with the condition can lose the ability to sit, crawl, walk, swallow, and eventually breathe.
Unlike conventional treatments that slow the disease’s progression, the administered therapy targets its root cause — delivering a functional copy of the missing SMN1 gene directly into the body, enabling the production of the protein motor neurons need to survive.
Dr. Chaudhry Muhammad Mehtab Iqbal, Division Chief of Paediatric Neurology at Tawam Hospital, led the procedure with the support of a multidisciplinary team that included genetics specialists, pharmacists, physiotherapists, and infusion unit staff. “This case represents a breakthrough in paediatric neurology and genetic medicine,” Dr. Iqbal said. “The administration of gene therapy for SMA is complex and requires careful planning, technical expertise, and teamwork. I am proud of our multidisciplinary team and the institutional commitment that made this a success.”
The therapy was carried out in compliance with UAE regulatory and ethical requirements, with coordination spanning the hospital’s neurology, pharmacy, and administrative departments. Tawam Hospital is a subsidiary of SEHA, which operates under PureHealth, described as the largest healthcare group in the Middle East.
Dr. Iqbal added that the achievement reflects the hospital’s broader commitment to “delivering world-class care and hope to children and families affected by rare diseases.”
For the young patient, the successful administration opens the possibility of reaching developmental milestones that SMA would otherwise have foreclosed.
