For the first time in Bangladesh, gene therapy has been applied to treat the chronic neurological disease Spinal Muscular Atrophy (SMA). The National Institute of Neurosciences & Hospital (NINS) has reached a new milestone in medical science by applying gene therapy to a child for the first time in Bangladesh to treat this congenital disease. This gene therapy, curable and USFDA (United States Food and Drug Administration) approved, costs around BDT 22 crore for a single dose and has been provided free of charge by the multinational pharmaceutical company Novartis Bangladesh.
Spinal Muscular Atrophy (SMA) is a rare and complex congenital disease of the nervous system caused by genetic disorder. The muscles of children suffering from this disease continue to weaken. As a result, the children cannot sit or stand. But they possess normal intelligence. Later, the affected children die due to respiratory complications. Every year, many children die in the world because of the lack of treatment for this disease. This disease usually destroys the nerve cells of the spinal cord of children. As the nerve cells that control the muscles are damaged, the muscles become weak. Most people in Bangladesh are not aware of this disease. The newborns suffering from this disease become very weak and gradually suffer from congenital heart disease.
But the silver lining is that for the first time in Bangladesh, it has been possible to apply the treatment of this rare disease with the initiative of Novartis and the support of the National Institute of Neurosciences & Hospital (NINS).
The Global Managed Access Program (GMAP) is designed to provide a feasible pathway for patients. Under this, patients who fulfill the required eligibility are selected. Treatment is then completed in accordance with applicable local laws and regulations.
A Bangladeshi child has been selected for this treatment under the afore-mentioned GMAP program. As the drug is prepared by taking into account each patient’s genetic characteristics and various other physical factors, which is a very complicated process, the treatment is very expensive. The medical cost of this gene therapy is unaffordable for the common people. Through this humanitarian initiative of Novartis, new doors have opened up for treating patients of SMA in Bangladesh. So far, this medicine has been administered to 2300 patients worldwide.
