Today's Edition
ABU DHABI (ALETIHAD)
SEHA's Sheikh Khalifa Medical City (SKMC), a subsidiary of PureHealth, announced significant advancements in the treatment of Duchenne muscular dystrophy (DMD). It has successfully administered its second case of gene therapy for DMD to a six-year-old Emirati patient named Zayed, who has since shown significant improvement.
DMD is a severe condition that primarily affects boys, leading to muscle weakness and heart complications. Historically, treatments have been limited, and life expectancy has been significantly shortened. However, recent developments in gene therapy have brought new hope.
Consultant Paediatric Neurologist at SKMC, Dr. Omar Ismayl, who has been at the forefront of the latest developments, explained, “We now have sophisticated gene therapy techniques that involve replacing the defective gene with a normal one using a viral vector. This therapy has shown promising results, with muscle fibres and overall muscle function returning to normal.”
The gene therapy process involves using a viral vector, specifically an adeno-associated virus (AAV), to deliver a functional version of the dystrophin gene to muscle cells. This gene encodes a protein essential for maintaining muscle health. The therapy aims to slow or stop the progression of the disease, allowing patients to retain better motor function for longer.
Dr. Ismayl elaborated, “The viral vector is engineered to carry the therapeutic gene into the patient’s muscle cells. Once inside, the gene begins to produce the dystrophin protein, which helps protect muscle cells from damage. This innovative approach has the potential to transform the lives of children with Duchenne muscular dystrophy (DMD).”
“Such procedures require a high level of expertise in gene therapy, and we are proud to be considered a centre of excellence in this field,” Dr. Ismayl added.
Zayed's parents expressed their gratitude, saying, “We are incredibly thankful to Dr. Ismayl and the entire team at SKMC. This treatment has given our child a new lease on life, and we are hopeful for the future.”
Dr. Ismayl also shared his optimism, stating, “Seeing the positive impact of gene therapy on our patients is truly rewarding. We are committed to advancing this treatment to benefit more children with Duchenne muscular dystrophy (DMD).”
