Abu Dhabi’s First Gene Therapy for Duchenne Muscular Dystrophy at Sheikh Khalifa Medical City
The Department of Health in Abu Dhabi has introduced a groundbreaking gene transfer therapy for Duchenne muscular dystrophy (DMD) at Sheikh Khalifa Medical City (SKMC). This marks the first time the treatment, previously only available in the US, has been administered in the emirate.
On March 19, 2024, a multi-disciplinary team at SKMC successfully administered the therapy to the first DMD patient in Abu Dhabi. The patient, a UAE national, received the treatment under the supervision of a specialized medical team led by a consultant pediatric neurologist.
DMD is a severe genetic disorder that causes muscle degeneration and weakness. The new treatment, delandistrogene moxeparvovec, is a one-time injection that delivers functional dystrophin genes into the patient’s cells. This helps the cells produce dystrophin protein, crucial for muscle function, thereby improving muscle strength and quality of life.
The therapy is available for children aged four to five with specific DMD gene mutations, but not for those with deletions in exon eight and/or exon nine of the DMD gene.
Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health, said, “This medical milestone underscores Abu Dhabi’s continuous dedication to adopting world-class healthcare innovations.”
Mohamed ElShaarawy, General Manager for Roche Pharmaceuticals UAE, expressed pride in collaborating with the Department of Health to enhance healthcare accessibility and quality.
Dr. Asma Ibrahim Al Mannaei, Executive Director of the Research and Innovation Centre at the Department of Health, highlighted the potential for valuable research opportunities and the establishment of a national genetic registry by the Emirati Genome Programme.
Dr. Omar Ismayl, Head of Pediatric Neurology at SKMC, emphasized the commitment to providing advanced treatments and technologies, aligning with PureHealth’s mission to unlock the science of longevity.
DMD primarily 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.
Doubts Revealed
Abu Dhabi -: Abu Dhabi is the capital city of the United Arab Emirates (UAE), a country in the Middle East.
Gene Therapy -: Gene therapy is a medical treatment that involves changing the genes inside your body’s cells to treat or stop disease.
Duchenne Muscular Dystrophy (DMD) -: Duchenne Muscular Dystrophy is a genetic disorder that causes muscles to become weak and damaged over time.
Sheikh Khalifa Medical City -: Sheikh Khalifa Medical City is a big hospital in Abu Dhabi where people go to get treated for various health problems.
Department of Health in Abu Dhabi -: The Department of Health in Abu Dhabi is a government organization that looks after the health and well-being of people living in Abu Dhabi.
Gene Transfer Therapy -: Gene transfer therapy is a type of gene therapy where new genes are added to a person’s cells to help treat a disease.
Delandistrogene Moxeparvovec -: Delandistrogene moxeparvovec is a special medicine used in gene therapy to help improve muscle function in people with Duchenne Muscular Dystrophy.
Emirate -: An emirate is a region or state that is ruled by an emir, and in this context, it refers to Abu Dhabi, one of the seven emirates in the UAE.
Dr. Noura Khamis Al Ghaithi -: Dr. Noura Khamis Al Ghaithi is a doctor who is important in the medical field and has helped bring this new treatment to Abu Dhabi.
Mohamed ElShaarawy -: Mohamed ElShaarawy is another key person who has played a significant role in introducing this new gene therapy treatment in Abu Dhabi.
