Sidra fifth globally to administer gene therapy for DMD

Sidra Medicine, a member of Qatar Foundation, has become the first hospital in Qatar and the fifth hospital to administer Elevidys worldwide, a revolutionary gene therapy medication for treating Duchenne Muscular Dystrophy (DMD).DMD is the most common form of inherited muscle disorders. It primarily affects males, with a prevalence of approximately 1 in 3,500 live male births1. Children with DMD typically appear normal at birth, but between the ages of two and three, begin to exhibit developmental delays, particularly in their motor skills and experience difficulties with walking, talking, jumping, or climbing stairs.Elevidys is currently the only gene therapy medication available for treating DMD. It works by delivering a functional dystrophin gene into the muscle and heart cells. The process slows disease progression and enhances muscle function. The drug was secured through Ebn Sina Medical, a local agent for Roche Pharmaceuticals, which is responsible for making Elevidys. It was cleared by the U.S. Food and Drug Administration (FDA) in June 2024, for a wide range of DMD patients, including children aged four and older.Dr. Tawfeg Ben-Omran, the Division Chief of Genetics and Genomic Medicine at Sidra Medicine said: "We are one of the few hospitals in the region with an extensive gene therapy program, reflecting our commitment to providing innovative, safe and effective treatments for genetic and rare diseases. Our collaborative and multidisciplinary team, headed by Dr. Khalid Ibrahim, Senior Consultant Neurologist and Neuromuscular Specialist, work across diagnosis, medication, treatment, prevention, and research - all through the lens of precision medicine. Adding a treatment protocol for DMD is another milestone for Sidra Medicine, showcasing our leadership as an expert in the field of personalized medicine for children with genetic diseases."Sidra Medicine administered Elevidys to a young international patient from Lebanon who became the first recipient of the drug in Qatar.Sidra Medicine is in the process of preparing three patients from Qatar and two young international patients from Kuwait to receive the same drug.The gene therapy program under the Genetic and Genomic Medicine clinic at Sidra Medicine is dedicated to providing life-saving treatments for rare genetic disorders. Its multidisciplinary team of geneticists, neurologists, therapists, and pharmacists are committed to delivering comprehensive care. Our services extend to patients from across the region, including Kuwait, Iraq, Iran, Libya, and North Africa.