SEHA’s new targeted therapy heals 3-year-old boy’s rare neurological disease

ABU DHABI, TGO: Abu Dhabi Health Services Company (SEHA) has used a new genetic therapy to successfully treat a three-year-old Emirati boy suffering from Duchenne Muscular Dystrophy (DMD).

With the child facing difficulties with standing and walking, the parents brought the child to Sheikh Shakhbout Medical City (SSMC), one of the UAE’s largest hospitals for serious and complex care and a joint-venture partnership between SEHA and Mayo Clinic, for a diagnosis. After a series of comprehensive tests, doctors identified mutations in the child’s DMD gene and subsequently diagnosed with DMD, a rapidly progressive form of muscular dystrophy. With the diagnosis, the child was referred to Sheikh Khalifa Medical City (SKMC), a SEHA healthcare facility, to access life-changing treatment.

DMD is a genetic disorder that causes progressive skeletal muscular weakness in children. If left without proper treatment, it can progress to affect the cardiac muscles and cause children to lose their ability to walk and develop scoliosis. This disease can also shorten the child’s life expectancy. The new therapy – Amondys 45 – was provided for the first time in the UAE at SKMC to treat the child after being diagnosed with DMD.

Speaking about this new breakthrough, Dr. Omar Ismayl, Consultant Pediatric Neurologist at SKMC, said, “Thanks to the new therapy treatments available at SKMC, such as Amondys 45, we are able to give the child and his parents new hope. This particular medication, administered as an intravenous infusion, allows for the production of the functional form of dystrophin: the protein that plays a vital role in maintaining muscle strength and is dysfunctional in children with DMD. This is a much-needed breakthrough and will enhance quality of life for many children living with DMD.”

Dr. Ismayl added, “Thirty percent of children who are affected with DMD are reported as the first sufferers in their families, while 70 percent are hereditary with previous cases in relatives, especially from the mother’s side. This neurological condition is caused by abnormal mutations or deletion in the dystrophin gene, which produces the essential protein that allows muscles to continue to be healthy and functioning.

“Currently, SKMC has four types of genetic treatments available for the treatment of muscle dystrophy conditions, so it is important for parents to do regular screenings and check-ups if they notice any abnormalities in the child’s motor functions. This will help doctors to determine the appropriate gene therapy treatment.”

The latest treatments are available for young patients with DMD and other neurological and muscle dystrophy conditions seeking treatment at Sheikh Khalifa Medical City (SKMC).

Adding to this, Dr. Safa Al Mustafa, Acting CEO at SKMC, said, “SKMC continues to provide the latest innovative treatment options to children with complex and serious medical conditions. With the support of our world-class team and through providing the latest advances in gene-based treatments, we are further enhancing and setting new benchmarks for children’s healthcare in the UAE.”