Abu Dhabi Pioneers Gene Transfer Therapy For Duchenne Muscular Dystrophy

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

On 19 March 2024, SKMC, in collaboration with multi-disciplinary teams from DoH's Research and Innovation Center (RIC), successfully treated the emirate’s first DMD patient. The UAE National received this advanced therapy under the supervision of a specialised medical team led by a consultant paediatric neurologist. Previously, this treatment was only available in the United States.

Duchenne muscular dystrophy is a severe genetic disorder that causes muscle degeneration and weakness over time. Delandistrogene moxeparvovec, a one-time injection, addresses the root cause of DMD by delivering functional dystrophin genes into patients' cells. This treatment instructs cells to produce dystrophin protein, essential for muscle function, thereby enhancing muscle strength and improving patients' quality of life.

Duchenne muscular dystrophy results from variations in dystrophin protein crucial for muscle cell integrity. When these variations occur, muscle fibres deteriorate leading eventually fatal weakening due cardiac or respiratory failure. Research shows that DMD primarily affects males with an estimated one in 3,500-5,000 boys worldwide having an average life expectancy around 30 years.

The therapy is available to children aged four to five with confirmed mutations in the DMD gene. However, it cannot be given to children with deletions in exon eight and/or exon nine of the DMD gene.

Dr Noura Khamis Al Ghaithi, Undersecretary of DoH, remarked: "This significant milestone marks a pioneering achievement in healthcare innovation, highlighting Abu Dhabi's pivotal role in advancing global medical breakthroughs. Collaborating with partners to diagnose and effectively manage the treatment of DMD is of utmost importance. This proactive approach, both by DoH and its partners, underscores Abu Dhabi's readiness to administer cutting-edge medical treatments, contributing to the growth of our medical tourism sector and solidifying Abu Dhabi's position as a regional and global healthcare hub."

Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, stated: "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.

"We are delighted to collaborate closely with the DoH to further enhance healthcare accessibility and quality for all, going beyond traditional pharmaceutical boundaries, and exploring research, education, and community awareness. I look forward to us continuing to work together to amplify the positive impact of healthcare by leveraging our collective expertise and resources."

Dr Asma Al Mannaei, Executive Director of RIC at DoH said: "The availability of treatments like delandistrogene moxeparvovec not only improve patient outcomes but also creates opportunities for researchers to delve deeper into understanding and advancing therapeutic interventions. This contributes to the regional and global pool of medical knowledge.

"Estimating the incidence and prevalence of DMD has been challenging due to factors such as the sporadic nature of genetic testing. However, this is set to change in the future with the Emirati Genome Programme and the opportunity to establish a national registry. Delandistrogene moxeparvovec presents a unique opportunity to initiate real-world evidence generation for gene therapy and prepare a registry specifically tailored to DMD."

This achievement highlights Abu Dhabi’s commitment towards innovative healthcare solutions while contributing significantly towards global medical advancements.