The Genetics and Genomic division at Sidra Medicine has launched several programmes to offer life-saving genetic treatment and therapies for patients of all ages. The programmes provide comprehensive genetic services including clinical evaluation and risk assessment; genetic counselling; diagnosis and the advanced management for a variety of genetic disorders.
Dr. Tawfeg Ben-Omran, the division chief of Genetics and Genomic Medicine at Sidra Medicine, said: “Genomic and genetic medicine is the critical bind that connects patients with genetic conditions to access therapies and resources that are personalised to treat their condition. It plays an important role in determining the risks of developing certain genetic diseases as well as screening and treatment. Our collaborative and multidisciplinary work entails the diagnosis, treatment, prevention and research in genetic and genomic medicine with respect to precision medicine.”
One of the key gene therapy programmes established at Sidra Medicine, in partnership with the paediatric neurology division, 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 severe morbidity and early death.
Children with SMA are cared for by a multidisciplinary team featuring physicians 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 that was included in a clinical trial to evaluate the safety and effectiveness of a novel gene therapy to treat babies with SMA.
Following detailed published clinical findings by the SMA team at Sidra Medicine and Hamad Medical Corporation, Sidra Medicine was appointed as the lead treatment center in the Middle East and North Africa (Mena) region to administer Zolgensma, a lifesaving drug for treating SMA. The Novartis Gene Therapies 'Managed Access Programme' works with select partner hospitals to offer gene therapies to babies suffering from rare and life threatening neurological genetic diseases and who are from countries where treatment and medications are not available.
Dr Khaled Omer Ibrahim and Dr Mahmoud Fawzi, senior attending physicians at Sidra Medicine and lead neurologists 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 pioneering and life-saving genetic therapies for SMA patients of all ages. Not only have we saved the lives of several patients in Qatar, in fact, because of Sidra Medicine’s selection by Novartis as a Mena treatment centre for administering the novel SMA medication, we are now welcoming 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 programme has been heartwarming and inspiring.
"We are currently in the process of implementing preventative SMA measures in collaboration with Qatar’s Premarital Genetic Screening and Expanded Metabolic Newborn Screening Programmes. We look forward to continuing this journey of healthcare innovation and excellence to benefit patients from everywhere.”
 
 
Related Story