Medcare UAE delivers breakthrough intrathecal gene therapy for international SMA patients

UAE—Medcare Women & Children Hospital in Dubai has delivered a groundbreaking intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a three-year-old child who travelled from Turkey for treatment.

This achievement places Medcare among the first private hospitals worldwide to offer this advanced therapy to an older international patient, surpassing previous age and weight restrictions that limited access to such care.

SMA is a rare genetic neuromuscular disease that gradually weakens the muscles needed for movement, swallowing, and breathing, often leading to severe disability if left untreated.

The newly licensed one-time intrathecal therapy targets the underlying genetic defect by delivering a working copy of the SMN1 gene, which helps motor neurons function more effectively and slows disease progression.

By addressing the root cause rather than only managing symptoms, this approach offers eligible patients a chance at more stable motor function and improved day-to-day abilities.

Before receiving gene therapy, Hulus relied on intensive physiotherapy and hydrotherapy just to maintain basic mobility, and his family expected a lifetime of frequent injections to manage his condition.

The intrathecal procedure at Medcare provided an alternative that may reduce the need for ongoing injections and lessen the overall treatment burden on both the child and his caregivers.

His father, Mustafa, explained that although the family had invested years in rehabilitation, this therapy has given them renewed hope that their son can gain greater independence and enjoy a better quality of life.

Dr. Shanila Laiju, Group CEO of Medcare Hospitals & Medical Centres, described this development as a defining moment for SMA care in the region, particularly for families whose children were previously excluded from gene therapy due to strict eligibility criteria.

She emphasised that more patients can now access a revolutionary option that has the potential to significantly improve long-term outcomes when combined with ongoing rehabilitation and specialist follow-up.

She also highlighted that this milestone reflects Medcare’s sustained focus on supporting children with rare diseases through specialised teams, advanced infrastructure, and continuous investment in cutting-edge therapies.

Since 2020, Medcare has treated more than 180 children with SMA, receiving families from countries including Iran, Turkey, Nepal, Romania, Russia, Kazakhstan, Kyrgyzstan, Ukraine, Belarus, Lebanon, India, and Ethiopia.

This growing international patient base underscores Dubai’s emergence as a trusted global hub for medical tourism, especially for paediatric genetic services and complex neuromuscular care.

Many families continue to receive coordinated follow-up support from Medcare even after returning to their home countries, which helps sustain treatment benefits and monitor long-term outcomes.

Consultant pediatric neurologist Dr. Vivek Mundada noted that older and heavier patients can now benefit from this transformative therapy, marking an important shift in how SMA is managed across age groups.

According to him, care is moving beyond narrowly focusing on infant survival toward preserving mobility, independence, and respiratory function in older children and adolescents living with the disease.

This change allows clinicians to personalise treatment plans that combine gene therapy with physiotherapy, respiratory support, and orthopaedic care to help patients maintain function for as long as possible.

Medcare provides SMA services through a multidisciplinary model that brings together paediatric neurology, pulmonology, orthopaedics, rehabilitation, genetic counselling, and structured long-term follow-up.

This integrated approach enables teams to address complications early, optimise supportive therapies, and guide families through complex decisions about care at each stage of the condition.

The hospital has also developed pathways tailored for international patients, ensuring that pre-arrival assessments, in-hospital treatment, and post-treatment monitoring remain coordinated and efficient.

Aligned with the UAE’s broader vision for medical tourism and specialised care, Medcare, part of Aster DM Healthcare, continues to invest in technologies, dedicated units, and expert clinical teams to treat rare and complex conditions such as SMA.

By expanding access to intrathecal gene therapy and related advanced interventions, the hospital aims to provide timely, high-quality care to patients who may have limited or no access to similar treatments in their home countries.