The Department of Health – Abu Dhabi (DoH) recently made history by providing revolutionary gene transfer therapy for Duchenne muscular dystrophy (DMD) in the UAE capital. This treatment, conducted at Sheikh Khalifa Medical City (SKMC), marks a milestone as Abu Dhabi becomes the first location outside the US to offer this therapy. The successful treatment of the emirates’ first DMD patient was carried out on March 19 this year, reflecting Abu Dhabi’s continuous growth as a leading destination for healthcare and life sciences on a global scale. The Emirati patient received the gene therapy under the supervision of a specialized medical team led by a consultant pediatric neurologist.
DMD is a genetic neuromuscular disorder characterized by the gradual degeneration and weakening of muscles. The gene therapy delandistrogene moxeparvovec is a one-time injection that addresses the underlying cause of DMD by delivering functional dystrophin genes into the patient’s cells, instructing them to produce dystrophin protein crucial for muscle function. This treatment has the potential to improve muscle strength and enhance the patient’s quality of life. However, the therapy is suitable for children between the ages of four and five with confirmed mutations in the DMD gene, excluding those with deletions in exon eight and/or exon nine of the gene.
Dr Noura Khamis Al Ghaithi, Undersecretary of DoH, emphasized the significance of this achievement in healthcare innovation, positioning Abu Dhabi as a hub for global medical breakthroughs. Collaborative efforts between DoH and its partners highlight the emirate’s readiness to administer cutting-edge medical treatments, furthering the growth of the medical tourism sector and establishing Abu Dhabi as a regional and global healthcare leader. Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, expressed pride in Roche’s commitment to patient-centric innovation and healthcare accessibility, particularly in the Middle East region.
Dr Asma Al Mannaei, the Executive Director of the Research and Innovation Centre at DoH, highlighted the opportunity presented by gene therapy treatments like delandistrogene moxeparvovec to enhance patient outcomes and contribute to advancing therapeutic interventions. The availability of such treatments supports further research and understanding of rare genetic disorders like DMD, aiding in the development of national registries and real-world evidence generation for gene therapy. Dr Omar Ismayl, Head of Paediatric Neurology at SKMC, applauded the introduction of gene therapy in Abu Dhabi, emphasizing SKMC’s commitment to providing advanced, evidence-based treatment and management to patients.
DMD primarily affects males and results from variations in the muscle protein dystrophin, leading to a breakdown of muscle fibers and gradual muscle weakness. With an average life expectancy of around 30 years, DMD is rare in females and affects approximately one in 3,500-5,000 boys worldwide. The successful implementation of gene therapy for DMD in Abu Dhabi showcases the emirate’s dedication to advancing medical technology and providing patients with access to state-of-the-art treatment options. This breakthrough marks a significant milestone in the field of healthcare innovation and underscores Abu Dhabi’s position as a global healthcare hub.
