UAE Hospitals Pioneer Gene Therapy for Spinal Muscular Atrophy Patients Across Age Groups
核心洞察
Medcare Royal Speciality Hospital (搜索) in Dubai became the world's first hospital outside the US to administer Itvisma (搜索) gene therapy to an adult SMA (搜索) patient, treating a 22-year-old Egyptian man.
Tawam Hospital in Abu Dhabi successfully delivered gene therapy to an 18-month-old Omani child with SMA (搜索), marking a major milestone in pediatric neurology.
The one-time gene replacement therapy addresses the root cause of SMA (搜索) by delivering a functional copy of the missing SMN1 (搜索) gene directly into cerebrospinal fluid.
Two leading UAE hospitals have achieved significant milestones in gene therapy for Spinal Muscular Atrophy (搜索) (SMA (搜索)), expanding treatment options for both pediatric and adult patients with this rare neuromuscular disease. Medcare Royal Speciality Hospital (搜索) in Dubai and Tawam Hospital in Abu Dhabi have successfully administered advanced gene therapies, positioning the UAE as a regional leader in genetic medicine.
Dubai Hospital Treats First Adult SMA Patient Outside US
Medcare Royal Speciality Hospital (搜索) made medical history by becoming the world's first hospital outside the United States to offer Itvisma (搜索), a newly licensed intrathecal gene therapy, to adult SMA (搜索) patients. The groundbreaking treatment was administered to a 22-year-old Egyptian patient who was diagnosed at 18 months and had been confined to a wheelchair for most of his life.
This achievement builds on Medcare's previous success, as the hospital had already become the first healthcare provider globally to administer Itvisma (搜索) to a four-year-old international SMA (搜索) patient last year. The expansion to adult patients represents a significant advancement, as gene therapy for SMA was previously restricted primarily to children under two years of age.
Dr. Shanila Laiju, Group CEO of Medcare Hospitals and Medical Centres, emphasized the transformative potential of this development: "We believe expanding SMA (搜索) treatment for adults can transform thousands of lives across the Middle East and beyond. The introduction of Itvisma (搜索) at Medcare builds on our pioneering role in treating older SMA patients, as recognised by Novartis."
Understanding SMA and Treatment Mechanism
SMA (搜索) is a rare genetic disorder that affects the nerves responsible for muscle movement, leading to progressive muscle weakness and loss of mobility. The condition impacts movement, breathing, and swallowing, occurring when the SMN1 (搜索) gene is missing or not functioning properly, leading to the deterioration of motor neurons over time.
The one-time gene therapy works by delivering a functional copy of the missing SMN1 (搜索) gene directly into the cerebrospinal fluid, addressing the root cause of the condition rather than merely managing symptoms. Dr. Vivek Mundada, clinical lead of Medcare's Gene Therapy Unit, explained the significance: "For years, curative treatment options were not available for adult SMA (搜索) patients. This opens new possibilities for many patients who were previously left without hope."
Abu Dhabi's Pediatric Success
Meanwhile, Tawam Hospital, part of SEHA (搜索) and a subsidiary of PureHealth (搜索), achieved its own milestone by successfully administering gene therapy to an 18-month-old Omani child diagnosed with SMA (搜索). The treatment was described as a major breakthrough in pediatric neurology and genetic medicine.
Dr. Chaudhry Muhammad Mehtab Iqbal, Division Chief of Pediatric Neurology at Tawam Hospital, led the treatment with support from a multidisciplinary team including pharmacists, therapists, and infusion unit specialists. "This case represents a breakthrough in pediatric neurology and genetic medicine," Dr. Iqbal stated. "The administration of gene therapy for SMA (搜索) is complex and requires careful planning, technical expertise and teamwork."
Global Impact and Patient Experience
The global burden of SMA (搜索) is significant, affecting 1 in 10,000 live births worldwide. Medcare has established itself as a regional treatment center, having treated over 190 SMA patients and 20 Duchenne Muscular Dystrophy (搜索) patients from the Middle East, Asia, and Europe.
Patient feedback has been positive, with the 22-year-old Egyptian patient noting: "The experience of taking the treatment was very positive and comfortable, with no pain felt around the injection area. I am glad that I don't have to take multiple injections like I used to before."
Advancing Regional Healthcare Capabilities
Both hospitals emphasized the complex nature of gene therapy administration, requiring careful planning, close coordination, and strict medical and ethical safety procedures. The successful treatments reflect the UAE's continued investment in advanced medical technologies and specialized healthcare services.
The multidisciplinary approach employed by both institutions demonstrates the integrated patient care model necessary for complex genetic therapies. These achievements position the UAE as a leader in genetic medicine within the region, offering hope to families affected by conditions once considered untreatable.