Today's Edition
ABU DHABI (ALETIHAD)
SEHA, a subsidiary of PureHealth, has revealed a significant medical milestone achieved at Tawam Hospital with 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.
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.
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.
"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," Dr Iqbal said.
He added, "It demonstrates Tawam Hospital's continued commitment to delivering world-class care and hope to children and families affected by rare diseases."
