Qatar- HMC implements innovative gene therapy to treat congenital spinal muscular atrophy – MENAFN.COM

(MENAFN - The Peninsula) 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 new gene therapy.

A team of doctors at Hamad General Hospital administered the innovative gene therapy to two pediatric patients diagnosed with the congenital disease, yesterday. A single dose of the newly introduced medication was injected into each patient and 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 $2.1m a dose and is being provided to Qatari pediatric patients free of charge.

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 Al Ansari extended gratitude to HH the Amir, the wise cabinet, and H E the Minister of Public Health, for their ongoing support of 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 QAR 500,000 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 will always remain committed to providing them with the best and latest treatments, added Dr Al Ansari.

Dr Tawfeg Ben-Omran, Senior Consultant, Pediatrics, and Head of Clinical and Metabolic Genetics said HMC becoming the second centre outside North America to offer innovative therapy is a significant achievement.

'Spinal muscular atrophy (SMA) is a genetic disorder and a leading cause of death in children under two years of age. The disease is characterized 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, said Dr Ben-Omran.

'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.

The proper dose of gene therapy has been successfully administered to two patients, noting the patients are 17 and 18 months old. Prior to the introduction of the new gene therapy, the two patients had been treated with repetitive spinal injections, a cumbersome procedure, but the new therapy is thought to be safer and more effective in the management of the disease.


Qatar- HMC implements innovative gene therapy to treat congenital spinal muscular atrophy - MENAFN.COM

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