Abu Dhabi [UAE], August 2 (ANI/WAM): The Department of Health – Abu Dhabi (DoH) has provided revolutionary gene transfer therapy for Duchenne muscular dystrophy for the first time in the emirate.
The treatment was carried out at the 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.
On March 19, 2024, in collaboration with multi-disciplinary teams at DoH’s Research and Innovation Centre (RIC), Sheikh Khalifa Medical City successfully administered the treatment to the emirates’ first DMD patient, following the treatment which was only available in the US.
The UAE National patient received the medication under the supervision of a specialised medical team led by a consultant paediatric neurologist.
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 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, 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 Ibrahim Al Mannaei, Executive Director of the Research and Innovation Centre at DoH, stated, “The availability of Delandistrogene moxeparvovec in Abu Dhabi has the potential to greatly enhance DMD patient outcomes and creates valuable research opportunities to further explore therapeutic interventions, enriching medical expertise regionally and internationally.”
Dr Al Mannaei added, “Measuring the incidence and prevalence of DMD has been challenging due to inconsistent genetic testing. However, this is set to change through the establishment of a national genetic registry by the Emirati Genome Programme. Delandistrogene moxeparvovec provides a unique opportunity to gather real-world evidence through gene therapy and the creation of a dedicated DMD registry.”
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. (ANI/WAM)
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