Medcare Expands Access to Breakthrough Intrathecal Gene Therapy for Spinal Muscular Atrophy, Treats First International Older Patient
Medcare Women & Children Hospital has reached a significant milestone in the treatment of Spinal Muscular Atrophy (SMA) by successfully administering a newly licensed intrathecal gene therapy to an international patient, marking a major advancement in care for older children and adults living with the rare genetic condition. The landmark procedure was performed on Hulus, a three-year-old child from Turkey, positioning Medcare among the first private healthcare providers globally to deliver this innovative therapy to an older, heavier international patient previously excluded from traditional gene therapy options.
This achievement represents a pivotal moment in the evolution of SMA treatment, expanding access beyond infants and addressing long-standing age and weight limitations that have historically restricted eligibility for gene therapy. By offering this newly approved, one-time intrathecal treatment, Medcare is helping reshape the SMA care landscape—both regionally and internationally—while reinforcing Dubai’s growing reputation as a global hub for advanced paediatric and genetic healthcare.
A Life-Changing Therapy Addressing the Root Cause of SMA
Spinal Muscular Atrophy is a rare, inherited neuromuscular disorder characterized by progressive muscle weakness and atrophy due to the loss of motor neurons in the spinal cord. The condition results from a defect or absence of the survival motor neuron 1 (SMN1) gene, which is essential for producing a protein that keeps motor neurons healthy and functional. Without sufficient SMN protein, affected individuals experience increasing difficulty with basic movements such as sitting, standing, walking, swallowing, and breathing.
While recent years have seen major progress in SMA treatment, early gene therapies were largely limited to very young patients, typically infants below a specific age and weight threshold. Older children and adults were often left with treatment options that required ongoing, lifelong administration—such as repeated intrathecal injections—to slow disease progression rather than address its genetic origin directly.
The newly licensed intrathecal gene therapy administered at Medcare represents a critical shift. Delivered directly into the spinal canal, the one-time treatment introduces a functional copy of the SMN1 gene, targeting the underlying genetic cause of SMA. By restoring the body’s ability to produce the essential SMN protein, the therapy offers the potential for long-term disease modification rather than continuous symptom management.
A New Chapter for Hulus and His Family
For Hulus and his family, the treatment marks a turning point after years of intensive rehabilitation and uncertainty. Prior to receiving gene therapy, his daily life revolved around frequent physiotherapy and hydrotherapy sessions aimed at maintaining muscle strength and mobility. While these interventions were essential, they demanded significant physical, emotional, and logistical effort, with no guarantee of lasting improvement.
Hulus’s father, Mustafa, described the journey as both challenging and hopeful. Despite the commitment to rehabilitation, the family knew that traditional therapies could only do so much. The availability of a one-time gene therapy—especially one that Hulus was now eligible to receive—offered renewed optimism.
“For years, we did everything possible to help our son through therapy and rehabilitation,” Mustafa shared. “This treatment has given us new hope—not just for improved mobility, but for a better quality of life and greater independence in the future.”
The successful administration of the therapy at Medcare underscores the hospital’s ability to manage complex, high-risk procedures through specialised expertise, advanced infrastructure, and comprehensive post-treatment monitoring.
Redefining SMA Care in the Region
“This is a defining moment for SMA care in the region,” said Dr. Shanila Laiju, Group Chief Executive Officer of Medcare Hospitals & Medical Centres. “Families whose children were previously ineligible for gene therapy due to age or weight restrictions now have access to a revolutionary treatment option. This breakthrough reflects Medcare’s long-standing commitment to supporting children with rare diseases through innovation, specialised clinical expertise, and world-class facilities.”
Dr. Laiju emphasized that the milestone is not only about introducing a new therapy but about expanding equity in access to life-changing treatments. By offering intrathecal gene therapy to older patients, Medcare is helping close a critical gap in SMA care—one that has left many families with limited options for years.
Shifting the Focus Beyond Infant Survival
Clinical experts note that the implications of this therapy extend far beyond a single patient or procedure. According to Dr. Vivek Mundada, Consultant Paediatric Neurologist at Medcare, the availability of gene therapy for older and heavier patients represents a fundamental shift in treatment goals for SMA.
“Historically, SMA care has focused on improving survival in infants diagnosed early,” Dr. Mundada explained. “While that remains crucial, this new therapy allows us to expand our focus toward preserving mobility, independence, and respiratory function in older patients. It transforms SMA management from a race against time in infancy to a comprehensive, lifespan-oriented approach.”
By intervening at later stages of the disease, clinicians aim to stabilize or improve motor function, slow further degeneration, and enhance overall quality of life—outcomes that were previously difficult to achieve in older SMA patients.
A Proven Track Record in SMA Treatment
Medcare’s latest achievement builds on years of experience in managing SMA cases from across the globe. Since 2020, the hospital has treated more than 180 children with SMA, welcoming families from countries including Iran, Turkey, Nepal, Romania, Russia, Kazakhstan, Kyrgyzstan, Ukraine, Belarus, Lebanon, India, and Ethiopia.
This extensive international patient base reflects both the hospital’s clinical reputation and Dubai’s strategic position as a destination for medical tourism. Families travel to Medcare seeking not only access to advanced therapies, but also coordinated care that addresses the full spectrum of medical, rehabilitative, and logistical needs associated with rare diseases.
A Multidisciplinary, Patient-Centered Model
Central to Medcare’s approach is a multidisciplinary care model that integrates expertise across multiple specialties. SMA patients at Medcare benefit from coordinated input from paediatric neurology, pulmonology, orthopaedics, rehabilitation medicine, genetic counselling, and specialised nursing teams. This collaborative framework ensures that treatment extends beyond the initial procedure to include long-term follow-up, monitoring, and supportive care.
For international patients, Medcare also provides dedicated coordination services to support families before, during, and after treatment—helping them navigate travel, accommodation, and continuity of care once they return home.
Supporting the UAE’s Vision for Advanced Healthcare
Medcare’s continued investment in cutting-edge therapies aligns closely with the UAE’s broader vision to position itself as a global leader in healthcare innovation and medical tourism. As part of Aster DM Healthcare, Medcare continues to expand its specialised units, attract top clinical talent, and adopt advanced technologies to address rare and complex medical conditions.
By introducing intrathecal gene therapy for SMA, Medcare not only enhances its own clinical capabilities but also contributes to the region’s growing reputation for delivering highly specialised, globally competitive healthcare services.
About
The successful treatment of Hulus marks the beginning of a new chapter in SMA care at Medcare. As more patients become eligible for intrathecal gene therapy, the hospital aims to broaden access further, refine clinical protocols, and contribute to global knowledge through continued research and collaboration.
For families affected by SMA—particularly those whose children were once considered too old or too heavy for gene therapy—this milestone represents something profound: possibility.
With innovation, expertise, and compassion at its core, Medcare is helping redefine what is achievable in the treatment of rare genetic disorders, offering renewed hope to patients and families around the world.
