The Department of Health - Abu Dhabi provides gene therapy for Duchenne Muscular Dystrophy for the first time in Abu Dhabi and outside the US

ABU DHABI, UAE, Aug. 2, 2024 /PRNewswire/ -- The Department of Health – Abu Dhabi (DoH), the regulator of the healthcare sector, has provided revolutionary gene therapy for Duchenne Muscular Dystrophy (DMD) for the first time in Abu Dhabi and outside the United States.

While previously the therapy was accessible only in the United States, this groundbreaking treatment will now also be offered at Sheikh Khalifa Medical City (SKMC), part of SEHA; a subsidiary of PureHealth, further cementing Abu Dhabi's position as a leading healthcare destination and life sciences hub globally. The treatment was approved by the U.S. Food and Drug Administration (FDA) in June 2023, and it was administered to an Emirati DMD patient by a specialist team led by a consultant paediatric neurologist from SKMC, in collaboration with the Department's Research and Innovation Centre on  March 19^th  2024.

DMD is a hereditary neuromuscular disorder that causes the muscles to gradually weaken  over time. Through this treatment, DoH and the SKMC medical team used Delandistrogene moxeparvovec; a one-time injection that has proven to be effective in addressing the underlying cause of DMD. The treatment can potentially transform the disease trajectory by delivering functional dystrophin genes into patient cells which help the body produce biologically active dystrophin protein for muscle function, improving muscle strength and the patient's quality of life. Following the FDA's announcement of expanding the Delandistrogene moxeparvovec treatment to children four and above, DoH remains committed to providing comprehensive care and support to affected patients and their families.

H.E. Dr. Noura Khamis Al Ghaithi, Undersecretary of Department of Health – Abu Dhabi (DoH), said: "This medical milestone underscores Abu Dhabi's continuous dedication to adopting world-class healthcare innovations that create a distinguished and holistic healthcare experience to all members of the community and beyond. We are committed to continuing to bring advanced preventative, diagnostics and therapeutic capabilities to the region, reinforcing Abu Dhabi's position as a leading healthcare and medical tourism destination".

Dr. Omar Ismayl, Head of Paediatric Neurology and Consultant at SKMC, said: "We at the Paediatric Neurology Department at SKMC are thrilled to use the gene therapy delandistrogene moxeparvovec for the first-time in Abu Dhabi. At SKMC, we are always committed to providing our patients with the most advanced, evidence-based management and treatment. This gene therapy underscores SEHA's commitment to providing access to the world's most advanced treatments and technologies, aligning with PureHealth's mission to unlock the science of longevity. It has the potential to change the unpleasant natural history and progression of this condition. This achievement would not have happened without the immense hard work and support from all the staff at SKMC and DoH."

DMD results from variations in a muscle protein called dystrophin, which normally maintains the integrity of muscle cells. When variations in this protein arise, muscle fibres break down, leading to a gradual weakening of the muscles that is eventually fatal due to cardiac or respiratory failure. Research indicates that DMD almost always affects males and is very rare in females. With an average life expectancy of 30 years, it affects an estimated one in 3,500-5,000 boys worldwide. 

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