Sidra Medicin’s gene therapy program saves lives

The Genetics and Genomic division at Sidra Medicine has launched several programs to offer life-saving genetic treatment and therapies to patients of all ages. The programs provide comprehensive genetic services, including clinical evaluation and risk assessment; genetic counseling; diagnosis and advanced treatment of a number of genetic disorders.

Dr. Tawfeg Ben-Omran, Head of Genetics and Genomic Medicine at Sidra Medicine, said: “Genomics and genetic medicine is the critical link that connects patients with genetic conditions to access therapies and resources tailored to treat their condition . It plays an important role in determining the risk of developing certain genetic diseases as well as screening and treatment. Our collaboration and interdisciplinary work includes diagnosis, treatment, prevention and research in genetic and genomic medicine with regard to precision medicine.”
One of the key gene therapy programs established at Sidra Medicine, in collaboration with the pediatric neurology department, is the treatment of children with spinal muscular atrophy (SMA), a neuromuscular disorder and a common fatal inherited disease resulting from a genetic mutation. If not treated in time, it can lead to serious morbidity and early death.
Children with SMA are cared for by a multidisciplinary team of doctors and nurses from clinical genetics and neurology and supported by rehabilitation therapies (including physiotherapy, occupational and speech therapy) and pharmacy services. Qatar is the only country in the region to be included in a clinical trial to evaluate the safety and effectiveness of a new gene therapy to treat babies with SMA.
Following detailed published clinical results from the SMA team at Sidra Medicine and Hamad Medical Corporation, Sidra Medicine was designated as the leading treatment center in the Middle East and North Africa (Mena) to administer Zolgensma, a life-saving drug for the treatment of SMA. Novartis Gene Therapies’ ‘Managed Access Programme’ works with selected partner hospitals to offer gene therapy to babies suffering from rare and life-threatening neurological genetic diseases who come from countries where treatment and medicine are not available.
Dr. Khaled Omer Ibrahim and Dr. Mahmoud Fawzi, chief attending physician at Sidra Medicine and senior neurologist in the SMA management team, said: “Thanks to the collaboration with the Ministry of Public Health and Hamad Medical Corporation and Sidra Medicine, Qatar has positioned itself as one of the few countries in the region to offer cutting-edge and life-saving genetic treatments for SMA patients of all ages Not only have we saved the lives of numerous patients in Qatar, in fact because of Sidra Medicine’s choice of Novartis as the Mena treatment center to administer the new SMA drug, we now welcome for patients from Iraq, Iran, Egypt and North Africa, as the therapy is not available in their countries.”
A patient family from Iraq had recently flown to Qatar to receive gene therapy for their young child. Dr. Tawfeg pointed out that the feedback from the patient families regarding the program has been heartwarming and inspiring.
“We are currently implementing SMA prevention measures in partnership with Qatar’s Premarital Genetic Screening and Expanded Metabolic Newborn Screening programs. We look forward to continuing this journey of healthcare innovation and excellence to benefit patients everywhere.”