Medcare in Dubai now offers a newly approved intrathecal gene therapy for Spinal Muscular Atrophy, expanding treatment to patients over age two and beyond the previous weight limits.
DUBAI: Medcare Royal Speciality Hospital in Al Qusais has become the first centre in the GCC — and among the first outside the US — to provide a newly approved intrathecal gene therapy for Spinal Muscular Atrophy (SMA), revolutionising treatment options for patients over two years old.
This advancement brings fresh hope to families of children who previously did not qualify for gene therapy due to age or weight restrictions. The new therapy, now licensed for intrathecal use in the UAE, enables treatment for older children, adolescents, and even adults living with this rare and life-threatening neuromuscular disorder.
Previously, gene therapy for SMA was limited to intravenous infusions suitable only for infants under two years or weighing less than 21kg. Many patients diagnosed later in childhood had no viable access to transformative treatment. The UAE’s approval and launch of this intrathecal therapy now addresses this unmet need and reinforces the country’s leadership in genetic medicine.
Dr. Shanila Laiju, Group CEO of Medcare Hospitals & Medical Centres, described the breakthrough as “a defining moment for SMA care.” She said, “For families who have exhausted options or were told their child was ineligible, this offers a renewed opportunity for improved quality of life.”
Since 2020, Medcare has treated over 180 children with SMA using gene therapy, drawing families from countries including Iran, Turkey, Nepal, Romania, India, and Ethiopia. Its growing international patient base highlights Dubai’s reputation as a trusted centre for rare disease care and medical tourism.
Dr. Vivek Mundada, Consultant Paediatric Neurologist at Medcare, emphasised the therapy’s impact: “This is a paradigm shift. We are now talking about helping older children and adults preserve independence — from standing and moving their arms to breathing more comfortably.”
SMA is caused by mutations in the SMN1 gene, leading to progressive muscle weakness. Around one in 40 people carry the faulty gene without symptoms. The new gene therapy uses a harmless viral vector to deliver a functional copy of SMN1, allowing the body to produce essential proteins for motor neuron survival. Unlike traditional treatments requiring frequent spinal injections, this is a one-time intervention that could modify the disease’s course.
Medcare follows a comprehensive, multidisciplinary approach in SMA care, with specialists across neurology, orthopaedics, pulmonology, genetic counselling, and long-term follow-up, even for international patients post-treatment.
Aligned with the UAE’s national vision of becoming a global hub for medical innovation, Medcare continues to invest in AI diagnostics, robotic surgery, and quaternary care. It aims to expand rare disease services and make critical treatments like SMA gene therapy more accessible to global patients.
