Sidra Medicine becomes first Qatari hospital to use revolutionary gene therapy medication
Sidra Medicine, a member of Qatar Foundation, made history as the first hospital in the country and the fifth in the world to administer Elevidys, 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 births. 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.
Sidra Medicine administered Elevidys to Chris El Kik, a young international patient from Lebanon who became the first recipient of the drug in Qatar.
Chris is currently under observation by a dedicated team from the hospital, who are closely monitoring the drug’s effectiveness. He and his family are also being provided with crucial outpatient services including child-life, nutrition, physical and mental health support.
He is expected to be in Qatar for several weeks. Since DMD takes years to manifest, the treatment protocol will take time, as his progress has to be regularly monitored.
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 US 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’re one of the few hospitals in the region with an extensive gene therapy programme, reflecting our commitment to providing innovative, safe, and effective treatments for genetic and rare diseases.
“Our collaborative and multidisciplinary team, headed by Senior Consultant Neurologist and Neuromuscular Specialist Dr Khalid Ibrahim, 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 personalized medicine for children with genetic diseases.”
“Our hospital has the capabilities and expertise to handle the extensive treatment protocols required for a drug like Elevidys. To illustrate, we were the first hospital in the region to administer it and care for Chris, an international DMD patient, whose family traveled from Lebanon to Qatar.”
“Through collaborations with international pharmaceutical partners like Roche Pharmaceuticals, Sidra Medicine continues to demonstrate its ability to offer the most advanced treatments to patients from all over the region and beyond,” said Dr Ben-Omran.
Dr. Ahmed Tawfik, the Head of Roche Pharmaceuticals - Gulf Cluster, said: "We’re thrilled to celebrate this momentous occasion, which underscores the immense value of innovation and highlights our unwavering commitment to providing hope to DMD patients. Together, we’re paving the way for a brighter future, where groundbreaking therapies offer renewed possibilities and improved quality of life for those affected by this challenging condition.”
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 programme under the Genetic and Genomic Medicine clinic at Sidra Medicine is dedicated to providing life-saving treatments for rare genetic disorders.
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