Babies as young as 5-weeks-old suffering from lethal spinal muscular atrophy, a common cause of death among children, can be safely treated with nurs Iversen, a newly discovered novel drug that may stop the progress of the said disease. The clinical trial of the new drug showed improved survival, slow development of the sickness and in few cases displayed notable improvement in the muscle function, as per the research. The new drug is presently being reviewed by the US Food and Drug Administration (FDA) for approval.
Spinal muscular atrophy or also called Werdnig-Hoffmann disease is a hereditary condition that causes extreme muscle weakness in babies and children. According to statistics, it affects almost 250 infants each year in the United States. Up to now, scientists have not found any cure for the disease. However a new clinically tested novel drug called nurs Iversen showed exceptional promise.
"With businesses, these infants are not only living longer but living better," Eureka Alert quoted Richard S. Finkel, MD, chief of neurology at Nemours Children's Hospital in Orlando, Fla and lead author of the research, emphasizing SMA is no longer death threats for babies. "This treatment is by no means a cure, but it is more than we've ever been able to offer these families before," he added.
For the study, 20 participating babies with SMA were injected with nursinersen treatments. The treatments were absorbed into the infants' nerve cells of the brain and spinal cord and then promote augmented production of an essential protein which is lacking in children with SMA.
Another good news is that majority of the participants was discovered to stop the progression of the disease. Babies treated with new cure showed improved motor function, enabled kids to gain abilities not present in spinal muscular atrophy type 1 which were rolling over, standing, sitting, and lastly improved survival.
Science Mag reported that out of 20 participating infants during the trial of the new drug, 13 are alive and kicking today, at between 2 and three years of age, while half of the untreated babies with this kind of disease typically die or end up on ventilators before they reach their first years. Due to the promising results of the new treatment, both European Medicines Agency and US FDA have included the drug at their fast track courses for proper drug approval.
