Fakeeh University Hospital sets new benchmarks in advanced gene therapy

Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialised treatments for rare genetic and neuromuscular disorders while reinforcing Dubai’s growing reputation as a centre for world-class tertiary care.

Among its landmark achievements, the hospital became the first facility in the region to administer Duvyzat (givinostat), an advanced therapy used in the management of Duchenne muscular dystrophy (DMD). DMD is a rare inherited neuromuscular disorder that primarily affects young boys, leading to progressive muscle weakness, loss of mobility, and serious cardiac and respiratory complications. Early and advanced intervention is critical to slowing disease progression and improving both survival and quality of life.

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In another major advancement, Fakeeh University Hospital has successfully administered one of the world’s first intrathecal gene therapies for spinal muscular atrophy (SMA), known as ITVISMA. SMA is a severe genetic condition characterised by degeneration of motor neurons, resulting in progressive muscle weakness, impaired breathing, and loss of motor function. Gene-based therapies have transformed outcomes for affected children, particularly when delivered early within specialised, multidisciplinary care environments.

Over the past 12 months, Fakeeh University Hospital has provided gene therapy–based treatments to children from multiple countries, with consistently positive clinical outcomes. Families have travelled internationally to Dubai to access these services, drawn by the hospital's specialised expertise, multidisciplinary care model, advanced infrastructure, and strong patient-centred approach.

Commenting on the programme’s progress, Arif Khan, director of the Gene Therapy Program and Consultant Paediatric Neurologist, said: "Our gene therapy program has been built to international standards, combining clinical excellence, rigorous safety protocols, and a deeply compassionate approach to care. The outcomes we are seeing, and the trust placed in us by families travelling from abroad, reinforce our commitment to continue expanding these services."

Dr Khan added that gene therapy is rapidly extending beyond neurology into areas such as haematology and inherited metabolic diseases. “Our long-term vision is to establish a comprehensive centre of excellence that supports the expanding role of gene therapy across specialties, positioning Dubai as a regional and international hub for advanced genomic medicine," he said.

Mohaymen Abdelghany, CEO of Fakeeh University Hospital, highlighted the hospital’s contribution to the UAE’s medical tourism strategy. "Fakeeh University Hospital is proud to support the UAE’s vision of becoming a global destination for advanced medical care,” he said. “By attracting patients from around the world for highly specialised, tertiary-level treatments, we are contributing to Dubai’s leadership in healthcare innovation while offering families access to advanced therapies within a trusted, internationally connected healthcare ecosystem."

For more information, visit Fakeeh University Hospital’s official website: www.fakeeh.health/