Gene Therapy for Older Children with DMD

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Fahad, a 10-year-old boy, was part of the first cohort of older children to received gene therapy for Duchenne Muscular Dystrophy (DMD) at the Gene Therapy Clinic at Sidra Medicine.

In late 2024, the age restriction to treat older children with DMD with Elevidys was updated. Sidra Medicine become the first hospital outside of the United States of America, to administer Elevidys gene therapy to children over the age of six.

Fahad’s mother said: “Initially we were told that there was no cure for this condition given my son’s age, which was devastating. However, after the age restriction for Elevidys had been lifted, it was a huge relief that Fahad’s condition was indeed treatable. This was life changing.”

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 medication. With the help of the doctors and nurses, I also learned how to better care for Fahad and manage his condition.”

The Gene Therapy Clinic at Sidra Medicine directly addresses the urgent needs of children with DMD from across the region. By bringing this lifesaving care closer to home and expanding the age eligibility, we are fostering innovation in DMD treatment and transforming the lives of children with DMD in the Arab region.

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At Sidra Medicine, Elevidys has only been administered to carefully selected patients who met strict eligibility criteria, including approved age and weight ranges. As of 19 June 2025, Sidra Medicine is not administering Elevidys for non-ambulatory patients with Duchene Muscular Dystrophy (DMD). The treatment protocol will only be administered to ambulatory DMD patients, who undergo thorough pre and post assessment and follow-up.

The safety and well-being of all our patients is our top priority. If you have any questions about our DMD treatment protocol, please email: genetherapyteam@sidra.org.