DMD Program now available to children from across the region
22 October 2024, Doha, Qatar - Sidra Medicine, a member of Qatar Foundation, has become the first hospital outside of the United States of America, to administer Elevidys gene therapy to children over the age of six.
The expansion of the hospital’s Duchenne Muscular Dystrophy (DMD) program now provides hope for older children with the condition. This groundbreaking achievement was highlighted by the recent successful administration of Elevidys to four Qatari children between the ages of nine to eleven years old, as well as two children aged four and five from Qatar and Kuwait, respectively.
Dr. Iyabo-Tinubu Karch, Chief Executive Officer at Sidra Medicine said: “Our program's expansion directly addresses the urgent needs of children with Duchenne Muscular Dystrophy from across the region. By bringing this lifesaving care closer to home and expanding the age eligibility, we are fostering innovation in DMD treatment. Our experience in this field, has established Sidra Medicine as a trusted and experienced healthcare partner. This approach to care, offers hope to children for a chance at a brighter future.”
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 that primarily affects boys. While appearing normal at birth, male children with DMD typically exhibit developmental delays in motor skills between the ages of two and three. This can manifest as difficulties in walking, jumping and climbing stairs or with talking.
Dr. Tawfeg Ben-Omran, Division Chief of Genetic and Genomic Medicine at Sidra Medicine, said: “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 children with Duchenne Muscular Dystrophy in the Arab region. We are beginning a new era of treatment for DMD. I am very proud of our efforts in establishing and expanding this program, which speaks to the expertise of our team and our world-class services. It also reflects how Qatar is increasingly being positioned as a top choice for caring for children with complex and rare genetic diseases.”
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.
Fahad, a 10-year-old boy, was one of the children who received gene therapy for Duchenne Muscular Dystrophy at Sidra Medicine. His mother said: "Initially we were told that there was no cure for this condition given my son’s age, which was devastating. However, after meeting Dr. Tawfeg, he reassured us that the condition is indeed treatable since the age restriction for Elevidys had recently been updated to treat older children. This was life changing. Dr. Tawfeg explained the condition clearly and got Fahad onto the appropriate medication."
She continued: "We are grateful for the incredible care provided by the entire team at Sidra Medicine, from the medical staff to the support staff, who treated us with the utmost kindness. I would like to extend special thanks to Dr. Tawfeg, Dr. Fawzi, and Dr. Khalid for their exceptional support. It wasn’t just about the medicine. With the help of the doctors and nurses, I also learned how to better care for Fahad and manage his condition."
Dr. Mahmoud Fawzi, Senior Attending Pediatric Neurologist at Sidra Medicine said: “We are pleased to report that all the boys who were administered Elevidys have responded well to the medication without any reported side effects. We are in constant touch with the families in following up on their children’s aftercare to monitor their progress.”
Sidra Medicine made history in June 2024, by becoming the first institution in Qatar and the fifth hospital in the world, to successfully administer Elevidys to a child. The administration of the drug to its first DMD patient Chris El Kik, served as a catalyst for the expansion of Sidra Medicine's DMD program.
Dr. Khalid Omer Ibrahim, Senior Attending Pediatric Neurologist at Sidra Medicine said: “Our multidisciplinary team of experts deliver comprehensive care to patients from across the MENA region. Since the expansion of our DMD program, we are now receiving multiple inquiries from many families including those with older children who are seeking easier access to innovative gene therapy treatments.”
At Sidra Medicine's Gene Therapy Clinic, we are dedicated to providing life-altering gene therapy for rare genetic disorders. If you based overseas, and want to inquire about our cutting-edge DMD program for your child, please contact International.services@sidra.org.