{"id":673,"date":"2016-10-27T16:12:24","date_gmt":"2012-10-27T16:12:24","guid":{"rendered":"http:\/\/www.viamedic.com\/blog\/?p=673"},"modified":"2012-10-27T16:12:24","modified_gmt":"2012-10-27T16:12:24","slug":"new-drug-may-help-boys-with-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/www.edrugstore.com\/blog\/current-health-news\/new-drug-may-help-boys-with-muscular-dystrophy\/","title":{"rendered":"New Drug May Help Boys With Muscular Dystrophy"},"content":{"rendered":"
\"\"
Children with muscular dystrophy remain hopeful that symptoms of this early onset disease can perhaps be remedied. (Photo by RON SOMBILON MEDIA, ART and PHOTOGRAPHY on Flickr.)<\/figcaption><\/figure>\n

This new drug appears to improve muscle function in boys<\/a> with muscular dystrophy, which is exciting because usually fighting muscular dystrophy means trying to slow the loss of muscle function. Once the decline begins, it usually never improves.<\/p>\n

The sudden and totally unprecedented reversal of this pattern in a test group of boys is \u201cincredibly promising\u201d according to Lee Sweeney, chairman of physiology at the University of Pennsylvania\u2019s Perelman School of Medicine. Lee Sweeney wasn\u2019t involved in the study, but he says the results are not normal at all.<\/p>\n

Muscular Dystrophy Treatment in America<\/strong>
\nThe most common type of muscular dystrophy is Duchenne muscular dystrophy, which affects about 13,000 boys in the United States. Because the disease is caused by a defect on the X chromosome, girls rarely get Duchenne muscular dystrophy — girls have a second copy of their X chromosome.<\/p>\n

The medicine, which is called eteplirsen, is targeted at a specific 13% of boys with Duchenne muscular dystrophy. This 13% has muscular dystrophy because of a specific mutation — one that can be corrected by eteplirsen, which tells the body skip a part of the dystrophin gene called exon 51.<\/p>\n

Other drugs are being developed to skip the genes exon 45 and exon 50, which will have the potential to help 9% and 5% of muscular dystrophy patients, respectively.<\/p>\n

\"\"
Research for muscular dystrophy is making strides, but the FDA approval process can be a frustrating obstacle for those seeking treatment. (Photo by Muscular Dystrophy Canada on Flickr.)<\/figcaption><\/figure>\n

FDA Approval?<\/strong>
\nThe only uncertainty so far with eteplirsen is how long it will take to make it through FDA approval processes. The initial test group is an extremely small patient population — only 12 subjects in total. Many test groups are several hundreds of patients, and there are often several repeated stages of testing before a drug is approved.<\/p>\n

Many patients are hoping for a \u201csuper-fast regulatory approval\u201d for eteplirsen, but while the results are convincing, Dr. Sweeney, who worked on the drug, says that a quick approval may be \u201ctoo much to hope for.\u201d<\/p>\n

The unfortunate truth is that the FDA has patient risk liability to think of first and foremost, and the organization will probably not rush a drug through the approval process based on just one study. There\u2019s always the chance, though, as other drugs have been approved with similarly limited experimental evidence, such as Genzyme\u2019s Myozyme drug. This drug, too, was designed to treat an extremely rare disease.<\/p>\n","protected":false},"excerpt":{"rendered":"

This new drug appears to improve muscle function in boys with muscular dystrophy, which is exciting because usually fighting muscular dystrophy means trying to slow the loss of muscle function. Once the decline begins, it usually never improves. The sudden and totally unprecedented reversal of this pattern in a test group of boys is \u201cincredibly […]<\/p>\n","protected":false},"author":1,"featured_media":674,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_autoblog_aioseo_focus_keyphrase":"","_autoblog_aioseo_title":"","_autoblog_aioseo_description":"","footnotes":""},"categories":[18],"tags":[],"class_list":["post-673","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-current-health-news"],"acf":[],"aioseo_notices":[],"aioseo_head":"\n\t\t\n\t\n\t\n\t\n\t\n\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t