SEHA’s Tawam Hospital Administers Groundbreaking Gene Therapy for Spinal Muscular Atrophy in 18-Month-Old Patient
SEHA, a subsidiary of PureHealth, the largest healthcare group in the Middle East, has revealed a significant medical milestone achieved at Tawam Hospital: the successful administration of gene therapy for Spinal Muscular Atrophy (SMA) in an 18-month-old female Omani.
The case involved an 18-month-old Omani girl who was diagnosed with Spinal Muscular Atrophy (SMA). SMA is a rare but serious genetic condition that affects the nerves controlling muscle movement. Children with SMA gradually lose muscle strength, which can make it difficult for them to sit, crawl, walk, swallow, or even breathe, depending on the severity of the disease. Without treatment, SMA can worsen over time and can become life-threatening.
Spinal muscular atrophy (SMA) occurs due to the absence or dysfunction of the SMN1 gene, which is essential for maintaining the health of motor neurones that control muscle movement. When this gene is missing or does not function properly, these neurones progressively deteriorate, leading to gradual muscle weakness over time.
To treat this underlying problem, the patient was offered a one-time gene replacement therapy. This advanced treatment works by providing the body with a healthy copy of the missing SMN1 gene, allowing the nerves to produce the protein needed to function properly. Unlike traditional treatments that only slow down the disease, this therapy targets the root cause of SMA.
Gene therapy has transformed the way doctors treat SMA around the world. Instead of managing symptoms alone, it gives children the chance to gain strength, improve their movement, and prevent the condition from worsening. For many families, this treatment represents a new chapter filled with hope, especially for a condition that was once considered untreatable.
For this young girl, it has opened a new chapter—one filled with hope, progress, and the possibility of reaching milestones that once felt out of reach.
The gene therapy was facilitated by Dr. Chaudhry Muhammad Mehtab Iqbal, Division Chief of Paediatric Neurologist, while under the support of a multidisciplinary team, including pharmacists, therapists and infusion unit staff to coordinate the therapy delivery. Collaboration across their expertise allowed for a thoughtful and safe administration of the therapy, in an organised manner, but also aligned with regulations and ethical safety measures that must be in place.
The successful implementation of this gene therapy was made possible through the leadership and guidance of the Chief of Paediatrics, with the support of the Chief Medical Officer and the Chief Executive Officer of Tawam Hospital. This demonstrates Tawam Hospital’s commitment to clinical excellence, innovation and aligning patient care
Working together collaboratively across the hospital’s clinical departments, hospital administration, and support services, Tawam Hospital continues to provide leading-edge medical solutions rapidly and efficiently.
This instance shows how Tawam Hospital is making strides in cross speciality collaboration. The team-driven approach of the neurology, genetics, pharmacy, physiotherapy, and infusion teams, in partnership with Dr Iqbal, is demonstrative of the model of collaborative care that Tawam espouses and ties closely to Tawam’s focus on medical innovation and holistic patient care.
This case reflects clinical success for Tawam and showcases Tawam Hospital’s commitment to compassionate, patient-centred care. The care and attention provided to every part of the treatment was anxiety alleviating for the young patient, while optimising his quality of life and outcomes; values that Tawam Hospital fully adheres to when caring for patients and families facing life-limiting disorders.
Dr. Chaudhry Muhammad Mehtab Iqbal, Division Chief – Neurologist at SEHA’s Tawam Hospital, said: “This case represents a breakthrough in paediatric neurology and genetic medicine. 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. It demonstrates Tawam Hospital’s continued commitment to delivering world-class care and hope to children and families affected by rare diseases.”
This achievement further establishes Tawam Hospital’s in advanced paediatric care, led by shared vision and leadership from its senior medical and executive leadership teams. Tawam Hospital’s commitment to innovation, collaboration and compassionate care drives the advancement of the future of genetic and neuromuscular medicine across the UAE and the region.
