First gene therapy for Duchenne Muscular Dystrophy launched by Department of Health

The Department of Health Abu Dhabi (DoH) has achieved a significant milestone in the field of healthcare by introducing gene transfer therapy for the treatment of Duchenne muscular dystrophy (DMD) for the first time in the emirate. This groundbreaking advancement took place at Sheikh Khalifa Medical City (SKMC), a leading facility under SEHA, a subsidiary of PureHealth.

On March 19, 2024, Sheikh Khalifa Medical City, in collaboration with multi-disciplinary teams from DoH’s Research and Innovation Centre (RIC), successfully administered the revolutionary gene therapy to the UAE’s inaugural DMD patient. Previously only available in the United States, this treatment was overseen by a consultant pediatric neurologist.

DMD is a severe genetic neuromuscular disorder characterized by the progressive degeneration and weakening of muscles. The newly introduced therapy, Delandistrogene moxeparvovec, involves a one-time injection that targets the root cause of DMD. By delivering functional dystrophin genes into the patient’s cells, the therapy enables the production of dystrophin protein crucial for muscle function. This results in improved muscle strength and overall quality of life for the patient.

This treatment is specifically designed for children aged four to five with a confirmed DMD gene mutation. However, it is not suitable for children with deletions in exon eight and/or exon nine of the DMD gene. Dr. Noura Khamis Al Ghaithi, DoH’s Undersecretary, highlighted the significance of this medical advancement, emphasizing Abu Dhabi’s commitment to integrating cutting-edge healthcare innovations and enhancing the healthcare experience for communities.

Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, expressed the company’s dedication to patient-centricity and improving health outcomes through collaboration with DoH. Dr. Asma Ibrahim Al Mannaei, Executive Director of DoH’s Research and Innovation Centre, emphasized the potential of Delandistrogene moxeparvovec to enhance DMD patient outcomes and offer valuable research opportunities.

Dr. Omar Ismayl, Head of Paediatric Neurology at SKMC, praised the introduction of gene therapy as a significant step in altering the progression of DMD. He noted that this development reflects SEHA’s commitment to providing access to world-leading treatments for various medical conditions.

DMD affects approximately one in 3,500-5,000 boys globally and is predominantly observed in males. The disorder leads to progressive muscle weakness and can be fatal by the age of 30 due to cardiac or respiratory failure. This breakthrough in medical technology showcases Abu Dhabi’s growing prominence in the global healthcare sector, offering hope and improved treatment options for patients with rare and complex conditions.

Overall, the successful administration of gene transfer therapy for DMD in Abu Dhabi represents a significant leap forward in the field of healthcare and highlights the emirate’s dedication to advancing innovative medical solutions for the benefit of its residents and beyond.