Dubai Hospital Achieves Breakthrough in Treating Adult Spinal Muscular Atrophy
Medcare Royal Specialty Hospital has become the first medical facility outside the United States to successfully treat an adult patient with Spinal Muscular Atrophy (SMA) using a newly licensed gene therapy, marking a major milestone in the global treatment of rare diseases.
The breakthrough involved a 22-year-old Egyptian patient who travelled to Dubai to receive the one-time treatment, offering new hope for adults living with the condition. SMA is a rare inherited disorder that causes progressive muscle weakness, affecting movement, breathing, and swallowing.
The therapy, developed by Novartis, works by delivering a functional copy of the missing SMN1 gene directly into the body, targeting the root cause of the disease. Until recently, such gene therapies were largely limited to infants and young children, leaving adult patients with limited treatment options.
Medical experts say the successful procedure represents a significant shift in the treatment landscape, opening access to advanced therapies for older patients who were previously excluded.
Dr Shanila Laiju, Group Chief Executive Officer of Medcare Hospitals, said the development could transform the lives of thousands of patients.
“Expanding access to advanced therapies for older patients is a meaningful step towards equity in care,” she said, adding that the hospital remains committed to delivering innovative treatments regardless of age or geography.
The procedure required a highly specialised, multidisciplinary medical team due to the patient’s complex medical history, including prior spinal surgery. Doctors involved in the treatment described it as a landmark achievement in gene therapy and a step forward in addressing rare genetic conditions.
Medcare has been at the forefront of gene therapy in the region, having treated more than 190 SMA patients and 20 patients with Duchenne muscular dystrophy since 2020. The hospital was also among the first private providers globally to administer the therapy to paediatric patients.
Health experts note that SMA affects approximately one in 10,000 births worldwide, making access to effective treatment critical for improving patient outcomes.
The development further strengthens Dubai’s position as an emerging global hub for advanced medical care, particularly in the treatment of rare and complex diseases.
