Families who were previously informed that their children had outgrown the eligibility for SMA gene therapy or were deemed not the right weight, now have renewed hope. Medcare Royal Speciality Hospital in Al Qusais has become among the first centres outside the USA to offer a newly licensed intrathecal gene therapy for Spinal Muscular Atrophy (SMA).
This treatment can now be administered to SMA patients well into their childhood and beyond. Approved after a thorough global assessment for intrathecal use, this therapy is tailored for children, teenagers, and adults, effectively removing the weight restrictions that have historically hindered access to gene therapy for this rare neuromuscular disorder. The milestone solidifies the UAE’s position at the forefront of cutting-edge genetic medicine and brings new hope to thousands of families worldwide.
SMA is a rare, inherited neuromuscular disorder that causes a weakening of the muscles essential for movement, breathing, and swallowing. If left untreated, it can be fatal for infants and can lead to significant disabilities in older children. Previous gene therapies required administration via intravenous infusion and were limited to young patients, usually limited to very young children, typically under two years of age or those below 21 kg weight threshold. This left many individuals diagnosed later in childhood or during their teenage years without a viable opportunity to access transformative treatment.
The new intrathecal gene therapy, now licensed in the UAE and immediately available at Medcare Royal Speciality Hospital, significantly expands eligibility beyond previous age and weight limitations, marking a pivotal shift in SMA treatment globally.
Commenting on this advancement, Dr. Shanila Laiju, Group CEO of Medcare Hospitals & Medical Centres, said, “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.”
“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,” she added.
Since 2020, Medcare has treated 180 children affected by SMA with gene therapy, including patients who travelled to the UAE from countries such as Iran, Turkey, Nepal, Romania, India, , and Ethiopia. A growing number of families from across the region and beyond are now choosing UAE for life-changing treatments, underscoring the city’s emergence as a trusted destination for advanced paediatric and genetic care as well as treatments for rare diseases. Dr. Shanila emphasised that it is both a responsibility and an honour for Medcare to expand treatment opportunities for SMA, aligning with the UAE’s broader vision of positioning Dubai as a global healthcare and medical tourism hub
From a clinical perspective, Dr
Vivek Mundada, Consultant Paediatric Neurologist at Medcare Royal Speciality Hospital, described the therapy as a transformative step forward in SMA care to the affected children over two years of age. “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.”
“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.
“SMA occurs when a child inherits two faulty copies of the SMN1 gene, one from each parent, leading to progressive muscle weakness. While approximately one in 40 people are carriers of the faulty gene without showing symptoms, affected children face severe physical limitations without timely intervention. The new gene therapy works by delivering a functional copy of the missing SMN1 gene using an inactive viral vector, allowing the body to produce the essential protein required for motor neuron survival,” Dr. Vivek explained.
“Unlike traditional SMA treatments that require repeated spinal injections over a lifetime, this therapy addresses the root genetic cause and represents a one-time, potentially disease-modifying intervention,” added Dr. Vivek, who has co-authored the recent clinical guidelines on gene therapy for SMA.
He further said that Medcare follows international standards of care for SMA patients, offering a fully integrated, patient-centred approach. This includes multidisciplinary expertise across neurology, pulmonology, orthopaedics, rehabilitation, and genetic counselling, along with long-term follow-up for international patients even after they return to their home countries.
According to Dr. Vivek, children who have received gene therapy at Medcare have shown encouraging improvements in mobility and physical function, significantly enhancing their quality of life.
Aligned with the UAE’s ambition to lead in medical innovation, Medcare continues to invest in advanced infrastructure, clinical talent, and next-generation technologies. Its focus on rare disease care is supported by specialised units, AI-enabled diagnostics, robotic surgical capabilities, and advanced quaternary care services, strengthening its position as a centre of excellence for complex genetic and neurological disorders.
As part of Aster DM Healthcare, Medcare remains committed to expanding access to advanced, high-quality medical care for both local and international patients. By combining clinical excellence with shorter wait times and comprehensive support services, Medcare continues to contribute to the UAE’s growing medical tourism sector, offering families timely and effective treatment options for critical and rare conditions such as Spinal Muscular Atrophy.