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Tribune News Network
Doha
HAMAD Medical Corporation (HMC) has become the second healthcare organisation outside the United States and Canada to treat children with congenital spinal muscular atrophy using a new gene therapy.
A team of doctors at Hamad General Hospital administered the innovative gene therapy to two paediatric patients diagnosed with the congenital disease recently.
A single dose of the newly introduced medication was injected into each patient. It will be followed by a prescribed therapeutic plan, as per the internationally approved protocol.
Qatar has become the second country outside North America to provide the new treatment, which is said to cost $2.1 million a dose and is being provided to Qatari paediatric patients free of charge.
Commenting on the new gene therapy being available to patients in Qatar, Dr Abdulla al Ansari, acting chief medical officer at HMC, said: “The application of this new gene therapy only six months after its approval by the Food and Drug Administration (FDA) in the United States reiterates HMC’s resolve and commitment to making the latest and most effective medications available to patients.”
Dr Ansari extended thanks and gratitude to His Highness the Amir, the wise Cabinet, and the Minister of Public Health, for their ongoing support to the healthcare sector and enabling HMC to provide its patients with the medications they need, regardless of cost.
“We wish a speedy recovery to these children. I would like to highlight the efforts of HMC’s clinical teams who provide care for, and treatment of, all patients with this condition. In the past, we have provided spinal muscular atrophy patients with the latest treatment available. The cost of that medication was QR500,000 per dose, and each patient required several doses. With the emergence of this new drug, which was approved by the FDA only 6 months ago, we immediately sought to obtain it for the benefit of our patients, despite its high price. HMC will always put its patients first and remain committed to providing them with the best and latest treatments”, added Dr Ansari.
Dr Tawfeg Ben-Omran, senior consultant, Paediatrics, and head of Clinical and Metabolic Genetics, said: “Spinal muscular atrophy (SMA) is a genetic disorder and a leading cause of death in children below two years of age. The disease is characterised by weakness and atrophy in the muscles used for movement, breathing and swallowing. Spinal muscular atrophy can be prevented through premarital screening and genetic testing before commencing IVF treatment for couples with a family history of the condition.”
“This highly advanced treatment involves the one-time intravenous administration of a healthy and functional gene which is delivered by a vector (a vehicle for delivering genetic material) to target the cause of SMA. The gene therapy improves the response of the child’s motor neurons. Dosage of treatment is determined based on the child’s weight”, added Dr Ben-Omran.
Dr Abdullah al Houthi, deputy chief, Paediatrics Department, Hamad General Hospital, said: “This morning, in the Pediatric Intensive Care Unit (PICU) at HGH, we administered the first dose of the innovative gene therapy to children with spinal muscular atrophy. The first dose was injected by a team that included Dr Khaled al Laithy, head of the Paediatric Intensive Care Unit at HGH and Dr Husam Ali, clinical pharmacist. Both patients are currently undergoing observation in the PICU.”
Dr Ali said the proper dose of the gene therapy medication for each pediatric patient is determined in close collaboration with the pharmaceutical company manufacturing the product.
Mohamed Ahmad al Meer, the father of one of the two paediatric patients who received the new treatment, expressed his sincere gratitude to Qatar’s leadership, commending the efforts of the country to enhance healthcare services and provide the latest treatments to all citizens, regardless of cost.
Doha
HAMAD Medical Corporation (HMC) has become the second healthcare organisation outside the United States and Canada to treat children with congenital spinal muscular atrophy using a new gene therapy.
A team of doctors at Hamad General Hospital administered the innovative gene therapy to two paediatric patients diagnosed with the congenital disease recently.
A single dose of the newly introduced medication was injected into each patient. It will be followed by a prescribed therapeutic plan, as per the internationally approved protocol.
Qatar has become the second country outside North America to provide the new treatment, which is said to cost $2.1 million a dose and is being provided to Qatari paediatric patients free of charge.
Commenting on the new gene therapy being available to patients in Qatar, Dr Abdulla al Ansari, acting chief medical officer at HMC, said: “The application of this new gene therapy only six months after its approval by the Food and Drug Administration (FDA) in the United States reiterates HMC’s resolve and commitment to making the latest and most effective medications available to patients.”
Dr Ansari extended thanks and gratitude to His Highness the Amir, the wise Cabinet, and the Minister of Public Health, for their ongoing support to the healthcare sector and enabling HMC to provide its patients with the medications they need, regardless of cost.
“We wish a speedy recovery to these children. I would like to highlight the efforts of HMC’s clinical teams who provide care for, and treatment of, all patients with this condition. In the past, we have provided spinal muscular atrophy patients with the latest treatment available. The cost of that medication was QR500,000 per dose, and each patient required several doses. With the emergence of this new drug, which was approved by the FDA only 6 months ago, we immediately sought to obtain it for the benefit of our patients, despite its high price. HMC will always put its patients first and remain committed to providing them with the best and latest treatments”, added Dr Ansari.
Dr Tawfeg Ben-Omran, senior consultant, Paediatrics, and head of Clinical and Metabolic Genetics, said: “Spinal muscular atrophy (SMA) is a genetic disorder and a leading cause of death in children below two years of age. The disease is characterised by weakness and atrophy in the muscles used for movement, breathing and swallowing. Spinal muscular atrophy can be prevented through premarital screening and genetic testing before commencing IVF treatment for couples with a family history of the condition.”
“This highly advanced treatment involves the one-time intravenous administration of a healthy and functional gene which is delivered by a vector (a vehicle for delivering genetic material) to target the cause of SMA. The gene therapy improves the response of the child’s motor neurons. Dosage of treatment is determined based on the child’s weight”, added Dr Ben-Omran.
Dr Abdullah al Houthi, deputy chief, Paediatrics Department, Hamad General Hospital, said: “This morning, in the Pediatric Intensive Care Unit (PICU) at HGH, we administered the first dose of the innovative gene therapy to children with spinal muscular atrophy. The first dose was injected by a team that included Dr Khaled al Laithy, head of the Paediatric Intensive Care Unit at HGH and Dr Husam Ali, clinical pharmacist. Both patients are currently undergoing observation in the PICU.”
Dr Ali said the proper dose of the gene therapy medication for each pediatric patient is determined in close collaboration with the pharmaceutical company manufacturing the product.
Mohamed Ahmad al Meer, the father of one of the two paediatric patients who received the new treatment, expressed his sincere gratitude to Qatar’s leadership, commending the efforts of the country to enhance healthcare services and provide the latest treatments to all citizens, regardless of cost.
