The newly licensed intrathecal gene therapy expands life-changing treatment options for children and adults affected by SMA

Dubai, United Arab Emirates: Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a three-year-old patient from Turkey. This milestone positions Medcare among the first private healthcare providers worldwide to offer this innovative treatment to an older international patient, overcoming previous age and weight limitations.

SMA is a rare neuromuscular disorder that progressively weakens muscles responsible for movement and breathing. This newly licensed one-time therapy addresses the root genetic cause by delivering a functional SMN1 gene.

For Hulus, whose daily routine earlier depended on intensive physiotherapy and hydrotherapy, the procedure from Medcare offered a promising alternative to lifelong injections. His father, Mustafa, shared that despite years of rehabilitation, the therapy has renewed hope for greater independence and quality of life for his son.

“This is a defining moment for SMA care in the region,” said Dr. Shanila Laiju, Group CEO of Medcare Hospitals & Medical Centres. “Families whose children were previously ineligible for gene therapy now have access to a revolutionary treatment that can significantly improve outcomes. This reflects Medcare’s long-standing commitment to children with rare diseases, supported by specialised expertise and advanced infrastructure.”

Since 2020, Medcare has treated more than 180 children with SMA, welcoming families from Iran, Turkey, Nepal, Romania, Russia, Kazakhstan, Kyrgyzstan, Ukraine, Belarus, Lebanon, India, and Ethiopia, reinforcing Dubai’s role as a strategic global hub for medical tourism and paediatric genetic care.

“Older and heavier patients can now benefit from transformative therapy,” said Dr. Vivek Mundada, Consultant Paediatric Neurologist. “This shifts SMA care beyond infant survival toward preserving mobility, independence, and respiratory function in older patients.”

Medcare delivers SMA care through a multidisciplinary model encompassing neurology, pulmonology, orthopaedics, rehabilitation, genetic counselling, and long-term follow-up for international patients.

Aligned with the UAE’s medical tourism vision, Medcare, part of Aster DM Healthcare, continues to invest in advanced technologies, specialised units, and clinical talent to provide timely, high-quality care for rare and complex conditions, including Spinal Muscular Atrophy.