Doha, Qatar: Sidra Medicine’s Genetic and Genomic Medicine clinic is providing life-altering gene therapy for rare genetic disorders. In a major achievement, the hospital became the first outside United States, to administer Elevidys gene therapy to four Qatari children with Duchenne Muscular Dystrophy (DMD), who were over the age of six.
DMD is a severe genetic disorder characterized by progressive muscle degeneration and weakness, primarily affecting boys. The treatment was initially available for children below the age of six years. With the expansion of Sidra Medicine’s DMD programme, the treatment now provides hope for older children with the condition.
“Sidra Medicine has recently successfully administrated of Elevidys to four children aged eight, nine, ten, and eleven years old,” Division Chief of Genetic and Genomic Medicine at Sidra Medicine, Dr. Tawfeg Ben-Omran told The Peninsula.
With an estimated prevalence of 1 in 3,500 live male births, DMD is one of the most severe forms of inherited and common muscular dystrophies. While appearing normal at birth, male children with DMD typically show developmental delays in motor skills between the ages of two and three. This can manifest as difficulties with walking, talking, jumping, or climbing stairs.
“Elevidys, a groundbreaking gene therapy developed by Roche Pharmaceuticals, is currently the only treatment of its kind for DMD. It works by introducing a functional dystrophin gene into muscle and heart cells, aiming to slow disease progression and improve muscle function,” said Dr. Ben-Omran.
In June 2024, Sidra Medicine made history by becoming the first hospital in Qatar and the fifth in the world to successfully administer Elevidys to a child. “The administration of this drug to Chris El Kik, served as a catalyst for the expansion of Sidra Medicine’s DMD programme,” said Dr. Ben Omran.
Since the expansion of the DMD programme, Sidra Medicine is now receiving multiple inquiries from many families from across the Middle East, including those with older children, who are seeking easier access to innovative gene therapy treatments.
“We are beginning a new era of treatment for DMD. By extending this lifesaving therapy beyond traditional age limitations, which were previously restricted to children under the age of six, Sidra Medicine is transforming the lives of DMD patients in the Arab region,” said Dr. Ben Omran.
