Sidra Medicine becomes first hospital in Qatar to administer revolutionary gene therapy medication

Gene therapy medication

Sidra Medicine, a member of Qatar Foundation, has made history as the first hospital in Qatar and the fifth hospital to administer Elevidys worldwide, 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 births1. 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.

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 U.S. 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 are one of the few hospitals in the region with an extensive gene therapy program, reflecting our commitment to providing innovative, safe and effective treatments for genetic and rare diseases. Our collaborative and multidisciplinary team, headed by Dr. Khalid Ibrahim, Senior Consultant Neurologist and Neuromuscular Specialist, 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 the field of personalized medicine for children with genetic diseases.” 

Dr. Ahmed Tawfik, the Head of Roche Pharmaceuticals - Gulf Cluster, said: "We are 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 are 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 administered Elevidys to Chris El Kik, a young international patient from Lebanon who became the first recipient of the drug in Qatar.

Dr. Ben-Omran added, "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. This not only exemplifies the trust in our care by his family but also the governments of Qatar and Lebanon as well as external partners who closely worked together in his case. 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.”

Chris is currently under observation by a dedicated team from the hospital, who are closely monitoring the drug's effectiveness and supporting him and his family 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.

Dr. Iyabo Tinubu-Karch, Chief Executive Officer at Sidra Medicine said: "Qatar's adoption of Elevidys solidifies its position as a leader in advanced healthcare and families seeking the best possible care for their children. We want to thank the Government of Qatar and the Ministry of Public Health, for expediting the life-saving drug being made available in the country. This accomplishment highlights our commitment to providing hope and world-class care for children with rare diseases, empowering them to live healthier and more fulfilling lives. We wish Chris all the best during his journey. With established protocols in place, we can confidently position ourselves as a trusted care provider and DMD gene therapy center for the GCC and MENA region.”

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 program under the Genetic and Genomic Medicine clinic at Sidra Medicine is dedicated to providing life-saving treatments for rare genetic disorders. Its multidisciplinary team of geneticists, neurologists, therapists, and pharmacists are committed to delivering comprehensive care. Our services extend to patients from across the region, including Kuwait, Iraq, Iran, Libya, and North Africa. For inquiries, please contact: International.services@sidra.org.