Abu Dhabi Debuts Groundbreaking Gene Therapy For Duchenne Muscular Dystrophy

The Department of Health – Abu Dhabi (DoH) has introduced gene transfer therapy for Duchenne muscular dystrophy (DMD) in the emirate. This groundbreaking treatment was administered at Sheikh Khalifa Medical City (SKMC), part of SEHA, a subsidiary of PureHealth. This development underscores Abu Dhabi's growth as a global healthcare and life sciences hub.

On 19th March 2024, SKMC successfully treated the first DMD patient in the UAE with this therapy. The procedure was conducted by a specialised medical team led by a consultant paediatric neurologist. Previously, this treatment was only available in the US.

Duchenne muscular dystrophy is a severe genetic disorder that leads to muscle degeneration and weakness over time. Delandistrogene moxeparvovec is a one-time injection designed to address the root cause of DMD. This therapy introduces functional dystrophin genes into the patient's cells, enabling them to produce dystrophin protein essential for muscle function.

This innovative treatment is available to children aged four to five with a confirmed mutation in the DMD gene. However, it cannot be administered to those with deletions in exon eight and/or exon nine of the DMD gene.

Dr Noura Khamis Al Ghaithi, Undersecretary of DoH, stated, "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."

Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, expressed pride in their collaboration with DoH. He said, "At Roche, we are immensely proud to be a global biotech company that is focused on innovation and driven by a commitment to patient-centricity. Our presence in the Middle East, and our mission to do now what patients need next, is a testament to our dedication to improving health outcomes in the region."

Enhancing Research Opportunities

Dr Asma Ibrahim Al Mannaei, Executive Director of the Research and Innovation Centre at DoH, highlighted the potential benefits of delandistrogene moxeparvovec. She noted that this treatment could significantly improve DMD patient outcomes while creating valuable research opportunities. Dr Al Mannaei added that establishing a national genetic registry through the Emirati Genome Programme would help gather real-world evidence and enhance genetic testing consistency.

Dr Omar Ismayl, Head of Paediatric Neurology at SKMC, shared his excitement about using delandistrogene moxeparvovec for the first time in Abu Dhabi. He 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."

Understanding Duchenne Muscular Dystrophy

DMD results from variations in dystrophin protein crucial for muscle cell integrity. These variations cause muscle fibres to break down over time, leading to progressive muscle weakening and eventual fatality due to cardiac or respiratory failure. Research shows that DMD predominantly affects males and is rare among females.

The average life expectancy for individuals with DMD is around 30 years. The disorder affects approximately one in 3,500-5,000 boys globally.

This achievement reflects SEHA's commitment to providing access to cutting-edge treatments and aligns with PureHealth's mission of advancing longevity science. Dr Ismayl emphasised that such progress wouldn't have been possible without the dedicated efforts of SKMC and DoH staff.

With inputs from WAM