The State of Qatar has made significant progress in treating children with hereditary spinal muscular dystrophy. The latest innovative gene therapy, costing $ 2.1 million, has been introduced at Hamad Medical Corporation.

This genetic disease is one of the most common causes of death of children under the age of two, and symptoms include weakness and atrophy of the muscles of the muscles, breathing muscles and swallowing. The patient is treated with only one dose of the new treatment and then completes the planned treatment program for such cases at Hamad Foundation according to an advanced international treatment protocol.

Qatar is the second to use this innovative gene therapy outside North America, free of charge for sick Qatari children, according to a statement from the Hamad Wasl Foundation.

Dr. Abdullah Al Ansari, Acting Chief Medical Officer at Hamad Foundation, said that the use of this new drug shortly after six months of its international adoption and use in the United States is a remarkable development in the Foundation's efforts to provide the latest treatments in the world to its patients, and keep abreast of global developments in the medical and therapeutic field.

Great support
Dr. Tawfiq Bin Omran, Senior Consultant and Head of the Department of Genetic Diseases and Metabolism at Hamad Foundation "This disease can be prevented by genetic testing before marriage as well as through genetic diagnosis before implantation through IVF technique, in case of a family history."

He explained that this highly sophisticated treatment depends on giving the proper gene through the vectors of muscle atrophy, by injecting the sick child intravenously only once, so that then works to improve the response of motor neurons of the child, and the dose of treatment is determined by the patient's weight.

For his part, d. Abdullah Al-Houthi, deputy head of the pediatric department at Hamad General Hospital, said that Qatar (thankfully) is one of the few countries that has started using this innovative gene therapy after its recent adoption by the US Food and Drug Administration.

For his part, said d. Khalid Al-Laithi, Consultant and Head of Pediatric Intensive Care at Hamad Hospital and Al Wakra Hospital, has successfully administered the new dose of gene therapy to two patients with hereditary spinal muscular dystrophy, aged 17 and 18 months. Before the emergence of the new highly developed treatment given to them, the patient had to take repeated doses of the drug in the spinal cord area at certain periods of life, so the importance of the new treatment comes from being more effective, safer and less Strain on the patient.