Medcare Royal Speciality Hospital in Al Qusais has begun offering a newly licensed intrathecal gene therapy for Spinal Muscular Atrophy (SMA), becoming one of the first centres outside the United States to provide the treatment.
The therapy expands access to gene treatment for SMA patients who were previously ineligible due to age or weight limits, allowing children, teenagers and adults to receive care.
Expanded SMA access
SMA is a rare inherited neuromuscular disorder that weakens muscles needed for movement, breathing and swallowing. Earlier gene therapies were delivered intravenously and were largely restricted to children under two years of age or below a specific weight threshold, leaving many older patients without treatment options.
The newly approved intrathecal therapy removes these limitations and is now available in the UAE following international regulatory reviews.
Hospital milestone
Medcare said the availability of the therapy marks a shift in SMA care, particularly for patients diagnosed later in childhood or adolescence.
“This is a defining moment for SMA care in the region and beyond. For families who have exhausted options or been told their child does not meet the criteria for gene therapy, this development offers something incredibly powerful – this development offers something incredibly powerful—a renewed opportunity for improved quality of life and functional outcomes,” said Dr. Shanila Laiju, Group CEO of Medcare Hospitals & Medical Centres.
She added, “The introduction of this therapy reflects Medcare’s long-standing commitment to advancing complex and specialised care in the region. She noted that Medcare has consistently focused on children with rare and challenging conditions, supported by highly specialised medical teams, advanced infrastructure, and a comprehensive care model designed specifically for women and children.”
Clinical perspective
Dr. Vivek Mundada, Consultant Paediatric Neurologist at Medcare Royal Speciality Hospital, said the treatment addresses a long-standing gap in care for older SMA patients.
“The therapy has undergone rigorous evaluation and regulatory approvals internationally, following rigorous international regulatory review, including evaluation by the U.S. Food and Drug Administration, before becoming available in the UAE,” he said.
“This milestone addresses a major unmet need in SMA care by expanding access to gene therapy for older and heavier patients, positioning Medcare and the UAE as a global referral destination for advanced SMA treatment. This is a genuine paradigm shift. We are no longer talking only about saving babies’ lives, but about helping older children and adults preserve their independence and them being able to stand, move their arms and even breathe more comfortably,” he added.
Treatment experience
Since 2020, Medcare has treated 180 children with SMA using gene therapy, including patients who travelled to the UAE from countries such as Iran, Turkey, Nepal, Romania, India and Ethiopia.
According to Medcare clinicians, patients who received gene therapy have shown improvements in mobility and physical function, with ongoing multidisciplinary care and long-term follow-up provided for both local and international families.
Press Release
