For the first time, UAE doctors treat rare disorder with gene therapy

The Department of Health – Abu Dhabi (DoH) has provided revolutionary gene transfer therapy for Duchenne muscular dystrophy (DMD), making the UAE capital the first to use this therapy outside the US.

The treatment was carried out at Sheikh Khalifa Medical City (SKMC), part of SEHA, a subsidiary of PureHealth, reflecting Abu Dhabi’s continual growth as a leading destination for healthcare and life sciences globally.

In collaboration with multi-disciplinary teams at the DoH’s Research and Innovation Centre (RIC), Sheikh Khalifa Medical City successfully treated the emirates’ first DMD patient, on March 19 this year.

The treatment was previously only available in the US.

The Emirati received the medication under the supervision of a specialised medical team led by a consultant paediatric neurologist.

What is DMD?
DMD is a debilitating genetic neuromuscular disorder characterised by the gradual degeneration and weakening of muscles. Delandistrogene moxeparvovec is a one-time injection that has been proven to address the underlying cause of DMD. This treatment holds the promise of potentially transforming the disease trajectory by delivering functional dystrophin genes into the patient’s cells. The treatment involves instructing the cells to produce dystrophin protein, which is crucial for muscle function, thereby improving muscle strength and improving the patient’s quality of life.

The delandistrogene moxeparvovec treatment is provided to children between the ages of four and five who have a confirmed mutation in the DMD gene. 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, said: “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, 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.

“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, the Executive Director of the Research and Innovation Centre 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.”

Dr Omar Ismayl, Head of Paediatric Neurology and Paediatric Neurology 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.