Ain Shams University provides treatment for the first Egyptian child to receive the most expensive medicine in the world, with a value of EGP 34 million

Under the patronage of Prof. Dr. Mahmoud El-Metini, President of Ain Shams University, the doctors of Ain Shams Specialized Hospital under the supervision of Prof. Dr. Nagia Ali Fahmy, Professor of Neurology and Director of the Musculoskeletal Unit at Ain Shams Medicine, by treating the first Egyptian child with spinal muscular atrophy disease, with the drug "Zolgensma", which is the most expensive drug in the world, with a value of $ 2.125 million, equivalent to 34 million Egyptian pounds.

Prof. Dr. Nagia stated that this drug is the first gene therapy of its kind in the world that is given to a patient via intravenous infusion and only once, and it is approved by the US Food and Drug Administration in May 2019.

She added that the drug-producing company offers 100 opportunities to obtain the drug free of charge in countries where the drug has not been registered, and sets conditions for choosing sick children, including that the child is no more than two years old and that the mutation is in the first gene, and accordingly the Muscular and Neurology Unit at Ain Shams Medicine advanced With 8 cases of injured children, the company selected the case of the child "Rayan" from the Alexandria governorate, who will be in his second year in a few days.

Dr. Nagia confirmed that Prof. Dr. Mahmoud El-Metini, President of Ain Shams University, provided full support to end the procedures for drug delivery, as he contacted the Minister of Finance and the President of the Medical Professions Union, to facilitate customs release and exemption from taxes and stamps estimated at 1.750 million pounds, and Novartis International Novartis Egypt contributed to providing training support to doctors on How to administer the treatment to the patient, and the procedures for transporting the treatment to Egypt at a temperature of (- 80) degrees Celsius were also facilitated.

She also explained that the injection process for the child was performed at Ain Shams Specialized Hospital, which includes faculty members and medical teams at the highest level in all specialties, noting that since the company was notified of the selection of the Egyptian child, each of Prof. Ayman Saleh, Vice President for Postgraduate Studies and Scientific Research, and Prof. Ashraf Omar, Dean of the Faculty of Medicine and Chairman of the University Hospitals Board of Directors, and Prof. Ali Al-Anwar, Executive Director of Ain Shams University Hospitals and Prof. Hani Aref, Head of the Department of Neurology and Prof. Walid Anwar, Director of Ain Shams Specialized Hospital, and his two deputies Dr. Muhammad Rizk and Dr. Hisham Anwar, to follow up training the medical staff on preparing the child for the injection process, training on the injection method and following it up in addition to the clinical pharmacy, as the medicine is preserved in a special way and under certain temperatures.

Regarding the rates of cure with this drug, Dr. Nagia Fahmy said that spinal muscular atrophy of the first and second types leads to the inevitable death of the child during the first two years of his life as a result of failure of breathing functions, and this drug has been clinically tested years ago and the first child who was injected is now five and a half years old, as the drug treats respiratory functions and motor impairment and puts The child is in the normal growth path, but the improvement is gradually taking place in stages, during which physiotherapy and pulmonary rehabilitative therapy are taken.

Regarding the Egyptian child "Rayan", Dr. Nagia said that the child will be injected with the genetic medicine and then will be followed up over a period of 3 months in all organic functions, through an integrated team of specialties of natural medicine, heart, genetics, chest and bones at Ain Shams Specialized Hospital, in addition to the team of the muscular and nerve diseases unit.

With regard to the possibility of making this drug available in Egypt, Prof. Dr. Nagia Fahmy said that there are hundreds of children with spinal muscular atrophy patients that this medicine will benefit them, but its costs are outside the capabilities of the state and people, pointing out that the Muscular and Neurological Diseases Unit, which was established 25 years ago in Ain Shams Medicine, was able to provide services that did not exist in diagnosis and treatment and the unit seeks For Egypt to be a center for research and clinical and clinical trials on genetic and hereditary diseases, which will provide treatment in Egypt at an affordable price, especially with the direction of pharmaceutical companies, including Egyptian ones, for research in this field.