{"id":363,"date":"2019-07-26T14:59:50","date_gmt":"2019-07-26T14:59:50","guid":{"rendered":"http:\/\/events.marketsandmarkets.com\/blog\/?p=363"},"modified":"2019-07-26T14:59:50","modified_gmt":"2019-07-26T14:59:50","slug":"gene-therapy-for-spinal-muscular-atrophy-approved-by-fda","status":"publish","type":"post","link":"https:\/\/events.marketsandmarkets.com\/blognew\/gene-therapy-for-spinal-muscular-atrophy-approved-by-fda\/","title":{"rendered":"Gene Therapy for Spinal Muscular Atrophy approved by FDA"},"content":{"rendered":"\n<p>A new treatment for a rare childhood disorder which cost\n$2.125 million for a single dose, one of the most expensive medicine available\nis approved by The US Food and Drug Administration.<\/p>\n\n\n\n<p>Spinal Muscular Atrophy, a condition driven by defects in\nthe SMN1 gene causing burdened babies lose muscle control, the medicine is\ndesigned to treat the disease. In the US each year about 400 babies is being\naffected by the illness and kills those with the most common type of the\ndisease in a couple of years. The gene therapy treatment uses genetically\nmodified viruses to deliver health copies of the SMN1 gene to patients\u2019 cells\nso they can produce a protein that enables the children to grow normally.<\/p>\n\n\n\n<p>In trial of the treatment, babies who got it by 6 months of\nage didn\u2019t have as serious muscle issues as those who didn\u2019t get the\nmedication. New-borns getting the drug after 6 months, didn\u2019t lose muscle\ncontrol, however they endured irreversible damage harm. According to Associated\nPress, those babies were the healthiest, who got the treatment early.<\/p>\n\n\n\n<p>David Lennon, President of AveXis, the company, owned by\nNovartis that developed the drug, called Zolgensma, said \u201cWe saw just\nremarkable results for these kids and it is only the second FDA-approved gene\ntherapy designed to treat genetic disorder.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>A new treatment for a rare childhood disorder which cost $2.125 million for a single dose, one of the most expensive medicine available is approved by The US Food and Drug Administration. Spinal Muscular Atrophy, a condition driven by defects in the SMN1 gene causing burdened babies lose muscle control, the medicine is designed to [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":365,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"inline_featured_image":false,"_mi_skip_tracking":false},"categories":[1],"tags":[],"_links":{"self":[{"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/posts\/363"}],"collection":[{"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/comments?post=363"}],"version-history":[{"count":1,"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/posts\/363\/revisions"}],"predecessor-version":[{"id":366,"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/posts\/363\/revisions\/366"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/media\/365"}],"wp:attachment":[{"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/media?parent=363"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/categories?post=363"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/events.marketsandmarkets.com\/blognew\/wp-json\/wp\/v2\/tags?post=363"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}