ABu Dhabi hospital administers gene therapy to child with rare disorder

Tawam Hospital, part of SEHA and a subsidiary of PureHealth, has successfully administered advanced gene therapy treatment to an 18-month-old Omani child diagnosed with Spinal Muscular Atrophy (SMA).

Officials described the case as a major medical milestone in the field of paediatric neurology and genetic medicine.

SMA is a rare genetic disorder that affects the nerves responsible for muscle movement. The condition causes gradual muscle weakness and can affect a child’s ability to sit, crawl, walk, swallow and breathe.

Doctors explained that the disease occurs when the SMN1 gene is missing or not functioning properly, leading to the deterioration of motor neurones over time.

One-time gene therapy

The child received a one-time gene replacement therapy designed to provide the body with a healthy copy of the missing SMN1 gene.

Unlike traditional treatments that focus on slowing the disease, the therapy aims to address the root cause of the condition.

Medical teams said the treatment offers children the chance to improve muscle strength and movement while helping prevent the disease from worsening.

Officials added that the therapy has brought new hope to families affected by SMA, a condition once considered untreatable.

Team effort

The treatment was led by Dr Chaudhry Muhammad Mehtab Iqbal, Division Chief of Paediatric Neurology at Tawam Hospital, with support from a multidisciplinary team that included pharmacists, therapists and infusion unit specialists.

Hospital officials said the successful treatment required careful planning, close coordination and strict medical and ethical safety procedures.

The hospital also highlighted the support provided by Tawam Hospital’s senior leadership and clinical teams in ensuring the therapy was delivered safely and efficiently.

“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,” he added.

Advanced paediatric care

Officials said the case reflects Tawam Hospital’s continued investment in advanced medical technologies and specialised healthcare services.

The hospital noted that the collaboration between neurology, genetics, pharmacy, physiotherapy and infusion teams reflects its approach to integrated patient care.

According to SEHA, the successful treatment further strengthens Tawam Hospital’s position in advanced paediatric and genetic care across the UAE and the wider region.

Hospital officials added that the focus remains on delivering innovative, compassionate and patient-centred care for children and families facing serious medical conditions.