Abu Dhabi DoH administers the innovative gene transfer for Duchenne muscular dystrophy for the first time

The Department of Health - Abu Dhabi (DoH) has achieved a groundbreaking milestone by administering innovative gene transfer therapy for Duchenne muscular dystrophy (DMD) for the first time in the emirate. This revolutionary treatment was conducted at Sheikh Khalifa Medical City (SKMC), part of SEHA, a subsidiary of PureHealth. This achievement underscores Abu Dhabi's rising prominence as a global leader in healthcare and life sciences.

How does it work?

Duchenne muscular dystrophy (DMD) is a severe genetic disorder that causes progressive muscle degeneration and weakness. The newly introduced treatment, Delandistrogene moxeparvovec, is a one-time injection designed to address the root cause of DMD. This therapy involves delivering functional dystrophin genes into the patient's cells, enabling them to produce dystrophin protein, essential for muscle function. By doing so, it significantly improves muscle strength and the overall quality of life for patients.

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

Why does it matter?

This medical breakthrough is a testament to Abu Dhabi's commitment to adopting cutting-edge healthcare innovations. Her Excellency Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, emphasized, "This medical milestone underscores Abu Dhabi's continuous dedication to adopting world-class healthcare innovations that create a distinguished and holistic healthcare experience for all members of the community and beyond."

The introduction of Delandistrogene moxeparvovec not only promises to transform the lives of DMD patients but also opens new avenues for research and therapeutic interventions.

Dr. Asma Ibrahim Al Mannaei, Executive Director of the Research and Innovation Centre at DoH, highlighted the potential research opportunities: "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."

The context

DMD is a debilitating genetic condition predominantly affecting males, with an average life expectancy of around 30 years. It is caused by variations in the dystrophin gene, which is crucial for maintaining muscle cell integrity. Without functional dystrophin, muscle fibers deteriorate, leading to severe muscle weakness and, ultimately, cardiac or respiratory failure.

This innovative treatment marks a significant leap forward in managing DMD, a condition that affects an estimated one in 3,500-5,000 boys worldwide.

Dr. Omar Ismayl, Head of Paediatric Neurology at SKMC, expressed the impact of this advancement: "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."

Moreover, the establishment of a national genetic registry by the Emirati Genome Programme is set to improve the consistency of genetic testing and measurement of DMD incidence and prevalence. This registry will provide valuable real-world evidence through gene therapy and enhance the overall understanding of DMD.

In collaboration with Roche Pharmaceuticals UAE, the DoH aims to further enhance healthcare accessibility and quality in the region. Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, noted, "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."

In conclusion, the administration of gene transfer therapy for DMD in Abu Dhabi represents a significant stride in medical innovation, offering new hope for patients and positioning Abu Dhabi at the forefront of global healthcare advancements.