The Department of Health – Abu Dhabi (DoH) has introduced groundbreaking gene transfer therapy for Duchenne muscular dystrophy (DMD), positioning the UAE capital as the first city outside the US to adopt this treatment. The therapy was administered at Sheikh Khalifa Medical City (SKMC), a part of SEHA and a subsidiary of PureHealth, showcasing Abu Dhabi’s ongoing ascent as a global leader in healthcare and life sciences. In partnership with multidisciplinary teams at the DoH’s Research and Innovation Centre (RIC), SKMC successfully treated the first DMD patient in the emirate on March 19 this year, a treatment previously exclusive to the US. The Emirati patient received the medication under the guidance of a specialized medical team led by a consultant pediatric neurologist.
DMD is a severe genetic neuromuscular disorder characterized by the progressive weakening and degeneration of muscles. Delandistrogene moxeparvovec, a one-time injection, has been proven to tackle the root cause of DMD by delivering functional dystrophin genes into the patient's cells. This treatment aims to instruct cells to produce dystrophin protein, essential for muscle function, thereby enhancing muscle strength and improving the patient’s quality of life. The delandistrogene moxeparvovec treatment is available for children aged four to five with a confirmed mutation in the DMD gene, excluding those with deletions in exon eight and/or exon nine.
Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, highlighted this milestone as a pioneering achievement in healthcare innovation, emphasizing Abu Dhabi’s role in advancing global medical breakthroughs. Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, expressed pride in Roche’s commitment to patient-centricity and innovation, looking forward to continued collaboration with the DoH to enhance healthcare accessibility and quality. Dr. Asma Al Mannaei, Executive Director of the Research and Innovation Centre at DoH, noted the treatment’s potential to deepen research into therapeutic interventions and contribute to global medical knowledge. Dr. Omar Ismayl, Head of Pediatric Neurology and Pediatric Neurology Consultant at SKMC, celebrated the use of gene therapy in Abu Dhabi, underscoring SEHA’s commitment to providing advanced treatments and technologies.
DMD arises from variations in the dystrophin protein, which normally maintains muscle cell integrity. When these variations occur, muscle fibers deteriorate, leading to progressive muscle weakness and ultimately fatal cardiac or respiratory failure. Research shows DMD predominantly affects males, with an average life expectancy of 30 years and an incidence of one in 3,500-5,000 boys worldwide.
