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The United Arab Emirates is rapidly emerging as a regional and international destination for advanced gene therapy, offering new hope to children affected by rare and life-threatening genetic conditions. At the forefront of this transformation is Fakeeh University Hospital, which continues to expand access to cutting-edge, gene-based treatments for complex neuromuscular disorders.
Spinal muscular atrophy (SMA) is a serious inherited neuromuscular disorder that affects approximately one in 6,000 to one in 11,000 live births worldwide. The condition is caused by a genetic mutation leading to degeneration of motor neurons, resulting in progressive muscle weakness, impaired breathing, and loss of motor function. Without early intervention, SMA remains one of the leading genetic causes of infant mortality.
Recent regulatory advancements in the UAE have enabled fast-tracked access to novel gene therapy treatments for SMA, offering patients the possibility of significantly improved outcomes. These therapies aim to address the underlying genetic cause of the disease rather than only managing symptoms, marking a major shift in how SMA is treated.
Clinical specialists report that eligible children receiving early gene-based intervention may experience improved muscle strength, enhanced mobility, and better long-term quality of life.
Fakeeh University Hospital has become a trusted center for families seeking advanced gene therapy solutions. Over the past year, the hospital has provided gene therapy-based treatments to children from multiple countries, achieving consistently positive clinical outcomes.
These treatments are delivered through a multidisciplinary approach, combining pediatric neurology, genetics, intensive care, and rehabilitation services to ensure comprehensive and personalized patient care.
In addition to SMA, Fakeeh University Hospital recently became one of the first hospitals in the region to administer Duvyzat (givinostat)—an advanced therapy used in the management of Duchenne muscular dystrophy (DMD).
DMD is another rare, inherited neuromuscular disorder that primarily affects young boys. It leads to progressive muscle degeneration, loss of mobility, and serious cardiac and respiratory complications. Early access to advanced therapies is critical in slowing disease progression, improving survival, and preserving quality of life.
The introduction of such therapies reflects the hospital’s commitment to precision medicine and innovation in rare disease management.
According to Dr. Arif Khan, the expanding trust placed in Fakeeh University Hospital by international families highlights Dubai’s growing reputation as a center for advanced genomic medicine.
“The outcomes we are seeing, and the trust placed in us by families travelling from abroad, reinforce our commitment to continue expanding these services. Gene therapy is rapidly extending beyond neurology into areas such as haematology and inherited metabolic diseases.”
He added that the hospital’s long-term vision is to establish a comprehensive center of excellence for gene therapy, supporting its application across multiple specialties and positioning Dubai as a regional and global hub for advanced genetic care.
With regulatory support, clinical expertise, and state-of-the-art infrastructure, Fakeeh University Hospital continues to lead the way in gene therapy and advanced treatments for rare genetic disorders. By focusing on early intervention, innovation, and multidisciplinary care, the hospital is transforming outcomes for children and families affected by complex inherited diseases.
