Dubai’s Medcare Hospital Treats First Adult SMA Patient Outside US with Gene Therapy
Medcare Royal Speciality Hospital has achieved a global medical milestone by becoming the first hospital outside the United States to administer a newly licensed intrathecal gene therapy to an adult patient with Spinal Muscular Atrophy (SMA).
The breakthrough treatment, Itvisma, was recently given to a 22-year-old Egyptian patient, marking a significant advancement in expanding access to gene therapy beyond early childhood.
A New Era for Adult SMA Treatment
SMA is a rare genetic condition that causes progressive muscle weakness, affecting mobility, breathing, and swallowing. Historically, gene therapy options have been largely limited to children under the age of two.
With the introduction of Itvisma, Medcare is now offering a one-time treatment for patients older than two years, opening new possibilities for adults who previously had limited therapeutic options.
How the Therapy Works
The gene therapy targets the root cause of SMA by delivering a functional copy of the missing SMN1 gene directly into the cerebrospinal fluid. This approach aims to restore essential protein production and improve neuromuscular function.
According to Dr. Vivek Mundada, the treatment represents a major shift in care: it addresses the disease at a genetic level rather than managing symptoms over time.
Multidisciplinary Expertise Behind the Procedure
The treatment was carried out by a specialised team at Medcare, including:
- Dr. Vivek Mundada
- Dr. Sagar Kawale
- Dr. Neha Shahane
- Dr. Ardalan Papari
Their collaborative effort highlights the complexity and precision required for advanced gene therapies.
Expanding Regional Access to Advanced Care
Dr. Shanila Laiju noted that expanding SMA treatment to adults could transform thousands of lives across the Middle East and beyond. She added that Medcare has already treated over 190 SMA patients and 20 patients with Duchenne Muscular Dystrophy (DMD) from multiple regions including the Middle East, Asia, and Europe.
The hospital had previously made headlines for administering the same therapy to a four-year-old international patient, further reinforcing its leadership in gene therapy innovation.
Patient Experience and Outlook
The patient, who had been wheelchair-bound for most of his life, described the procedure as comfortable and pain-free, highlighting the advantage of a single-dose treatment compared to repeated injections.
A Milestone for Global Healthcare
This development positions Dubai as a growing hub for advanced medical innovation, particularly in gene therapy and rare disease treatment. It also reflects the UAE’s broader commitment to bringing cutting-edge healthcare solutions to the region.
