Abu Dhabi: The Department of Health Abu Dhabi (DoH) has made a groundbreaking advancement in the treatment of Duchenne muscular dystrophy (DMD) by administering gene transfer therapy for the first time in the emirate.
The pioneering treatment was performed at Sheikh Khalifa Medical City (SKMC), a key facility under SEHA, a subsidiary of PureHealth.
On March 19, 2024, the 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 first DMD patient. Previously available only in the US, this treatment was provided under the expert supervision of a consultant pediatric neurologist.
DMD is a severe genetic neuromuscular disorder characterized by the progressive degeneration and weakening of muscles. The new therapy, Delandistrogene moxeparvovec, is a one-time injection proven to address the root cause of DMD. It delivers functional dystrophin genes into the patient’s cells, enabling the production of dystrophin protein essential for muscle function, thereby improving muscle strength and overall quality of life.
The treatment is specifically available for children aged four to five with a confirmed DMD gene mutation, however, it cannot be administered to children who have any deletions in exon eight and/or exon nine of the DMD gene.
Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH emphasized that, “This medical milestone reflects Abu Dhabi’s unwavering commitment to integrating cutting-edge healthcare innovations, enhancing the holistic healthcare experience for our community and beyond.”
Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE stated that, “Our collaboration with DoH is a testament to our dedication to patient-centricity and improving health outcomes. We are excited to explore new horizons in healthcare together, focusing on research, education, and community awareness.”
Dr. Asma Ibrahim Al Mannaei, Executive Director of DoH’s Research and Innovation Centre, highlighted the potential of Delandistrogene moxeparvovec to improve DMD patient outcomes and provide valuable research opportunities.
Dr. Omar Ismayl, Head of Paediatric Neurology at SKMC, praised the introduction of gene therapy and said that, “This therapy represents a significant step in changing the progression of DMD and reflects SEHA’s dedication to providing access to world-leading treatments.”
DMD, which affects approximately one in 3,500-5,000 boys globally and is almost exclusively seen in males, leads to progressive muscle weakness and is often fatal by the age of 30 due to cardiac or respiratory failure.
This breakthrough medical development signifies Abu Dhabi’s growing prominence in the global healthcare arena, offering new hope and improved treatment options for patients with rare and challenging conditions.