Provided by Department of Health – Abu Dhabi, innovative gene transfer therapy administered for Duchenne muscular dystrophy for first time in the Abu

The Department of Health – Abu Dhabi (DoH) has made a significant medical breakthrough by providing revolutionary gene transfer therapy for Duchenne muscular dystrophy (DMD) for the first time in the emirate. This landmark treatment was performed at Sheikh Khalifa Medical City (SKMC), a part of SEHA, under the umbrella of PureHealth. This advancement showcases Abu Dhabi’s growing reputation as a global leader in healthcare and life sciences.

On March 19, 2024, a multidisciplinary team from DoH’s Research and Innovation Center (RIC) and Sheikh Khalifa Medical City successfully administered the gene therapy to the first DMD patient in the emirate. This treatment, previously available only in the United States, was overseen by a specialized medical team led by a consultant paediatric neurologist. The patient, a UAE national, received the medication to address the debilitating effects of DMD, a genetic disorder characterized by progressive muscle degeneration and weakness.

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The therapy, known as delandistrogene moxeparvovec, is a one-time injection that delivers functional dystrophin genes into the patient's cells. These genes instruct the cells to produce dystrophin protein, which is essential for muscle function. This treatment has the potential to significantly improve muscle strength and enhance the quality of life for DMD patients.

Delandistrogene moxeparvovec is intended for children aged four to five who have a confirmed mutation in the DMD gene. However, it is not suitable for children with deletions in exon eight and/or exon nine of the DMD gene.

Her Excellency Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, emphasized the importance of this milestone, stating, “This significant achievement highlights Abu Dhabi’s pivotal role in advancing global medical breakthroughs. Collaborating with partners to diagnose and manage DMD is crucial. This proactive approach underscores Abu Dhabi’s readiness to administer cutting-edge medical treatments, contributing to the growth of our medical tourism sector and solidifying our position as a regional and global healthcare hub.”

Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, expressed pride in the collaboration, stating, “At Roche, we are focused on innovation and patient-centricity. Our mission to meet patient needs reflects our dedication to improving health outcomes in the region. We are delighted to collaborate with DoH to enhance healthcare accessibility and quality, exploring research, education, and community awareness together.”

Dr. Asma Al Mannaei, Executive Director of the Research and Innovation Centre at DoH, highlighted the broader impact of the treatment, saying, “The availability of delandistrogene moxeparvovec not only improves patient outcomes but also creates opportunities for advancing therapeutic interventions. This contributes to the regional and global pool of medical knowledge. The Emirati Genome Programme and a national registry will help better estimate the incidence and prevalence of DMD and generate real-world evidence for gene therapy.”

Dr. Omar Ismayl, Head of Paediatric Neurology and Consultant at SKMC, praised the introduction of the gene therapy, stating, “We are thrilled to use delandistrogene moxeparvovec for the first time in Abu Dhabi. SKMC is committed to providing the most advanced, evidence-based treatments. This gene therapy aligns with SEHA’s and PureHealth’s mission to offer world-class treatments and technologies. It has the potential to change the natural progression of DMD. This achievement is a result of the hard work and support from SKMC and DoH staff.”

Duchenne muscular dystrophy (DMD) results from mutations in the dystrophin gene, which is critical for maintaining muscle cell integrity. Without functional dystrophin, muscle fibers deteriorate, leading to severe muscle weakness and eventually fatal cardiac or respiratory failure. DMD primarily affects males, with an average life expectancy of around 30 years, impacting approximately one in 3,500-5,000 boys worldwide.