Sidra Medicine Groundbreaking Gene Therapy for Older Children. Doha, Qatar – Sidra Medicine, renowned for its cutting-edge advancements in Genetic and Genomic Medicine, has achieved a major milestone by becoming the first hospital outside the United States to administer Elevidys gene therapy to older children with Duchenne Muscular Dystrophy (DMD).
This life-changing therapy has been successfully provided to four Qatari children, all above the age of six, marking a new era in the treatment of this severe genetic disorder.
DMD is a rare but devastating condition that primarily affects boys, leading to progressive muscle degeneration and weakness. Typically, children with DMD begin showing symptoms of motor skill delays between the ages of two and three, with difficulties in walking, climbing stairs, and other physical activities.
While the condition was once primarily treated in younger children, Sidra Medicine has now expanded its DMD program, offering gene therapy to older patients, providing hope to families affected by this debilitating disorder.
A Landmark Achievement in Gene Therapy
In a historic achievement, Sidra Medicine became the first hospital in Qatar and the fifth in the world to administer the Elevidys gene therapy. This breakthrough therapy, developed by Roche Pharmaceuticals, introduces a functional dystrophin gene into the muscle and heart cells of patients with Duchenne Muscular Dystrophy.
The aim is to slow the progression of the disease and improve muscle function, offering a significant improvement in the quality of life for those affected.
The therapy was initially available only to children under six years of age. However, thanks to the expansion of the DMD program at Sidra Medicine, children aged eight, nine, ten, and eleven have now received the treatment.
According to Dr. Tawfeg Ben-Omran, Division Chief of Genetic and Genomic Medicine at Sidra Medicine, “The successful administration of Elevidys to these children has opened up a new era of treatment for DMD, providing hope to families not just in Qatar but across the Middle East.”
Importance of Early Detection and Expanded Treatment Options
With an estimated prevalence of 1 in 3,500 live male births, Duchenne Muscular Dystrophy is one of the most severe inherited muscular dystrophies. Despite appearing healthy at birth, boys with DMD often face developmental delays by the time they reach preschool age. These early warning signs include difficulty with motor skills like walking, jumping, and climbing stairs, which often lead to a diagnosis of DMD.
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Until recently, gene therapy treatments like Elevidys were only available for younger children, under the age of six. However, Sidra Medicine expanded program has made it possible for older children to access this revolutionary treatment. This expansion is a game-changer for families with older DMD patients who had previously been ineligible for gene therapy.
A New Era of Treatment for Duchenne Muscular Dystrophy
The administration of Elevidys in June 2024 to Chris El Kik, the first child in Qatar to receive this treatment, was a pivotal moment for Sidra Medicine. It not only highlighted the hospital leadership in genomic medicine but also served as the catalyst for the expansion of the DMD program.
As Dr. Ben-Omran pointed out, “The success of this treatment has drawn the attention of families from across the Middle East, many of whom are now inquiring about access to gene therapy for their children.”
The expansion of the program has garnered significant attention, with many families from across the region now seeking innovative gene therapy treatments at Sidra Medicine. The hospital’s pioneering efforts to provide access to these treatments for older children have made it a beacon of hope for those battling DMD in the Arab world.
Future of DMD Treatment at Sidra Medicine
By pushing the boundaries of age limitations for gene therapy, Sidra Medicine is transforming the landscape of DMD treatment in the Middle East. The hospital commitment to innovative healthcare solutions is reshaping the lives of DMD patients, offering them improved outcomes and a better quality of life. As Dr. Ben-Omran stated,
“We are beginning a new era of treatment for DMD, extending lifesaving therapies to older children who were previously ineligible. This is truly life-changing for patients and their families.”
The provision of Elevidys gene therapy at Sidra Medicine is not only a testament to the hospital’s world-class capabilities in genetic and genomic medicine but also underscores its dedication to improving patient care across the region. As more families seek treatment options, Sidra Medicine is poised to continue its leadership in offering lifesaving gene therapies for rare genetic disorders.
Conclusion
Sidra Medicine expansion of its Duchenne Muscular Dystrophy (DMD) program and its successful administration of Elevidys gene therapy to older children marks a significant advancement in the treatment of this devastating genetic disorder. With DMD affecting a significant number of boys in the Middle East, the hospital’ efforts are providing hope to families across the region.
By extending treatment options beyond traditional age limits, Sidra Medicine is at the forefront of improving the lives of DMD patients, offering a brighter future for those affected by this condition.
As the hospital continues to lead the way in genomic medicine, its impact on rare genetic disorder treatments will undoubtedly grow, bringing innovative gene therapies to even more patients and cementing Sidra Medicine‘s position as a global leader in healthcare innovation.