{"id":5314,"date":"2023-05-10T05:19:29","date_gmt":"2023-05-10T05:19:29","guid":{"rendered":"https:\/\/healthnewscentral.com\/?p=5314"},"modified":"2023-05-10T05:19:29","modified_gmt":"2023-05-10T05:19:29","slug":"child-diagnosed-with-deadly-mld-found-to-be-cured-for-the-first-time-in-history","status":"publish","type":"post","link":"https:\/\/healthnewscentral.com\/child-diagnosed-with-deadly-mld-found-to-be-cured-for-the-first-time-in-history\/","title":{"rendered":"Child Diagnosed With Deadly MLD Found To Be Cured For The First Time In History"},"content":{"rendered":"
\"\"
Medical News Today<\/figcaption><\/figure>\n

Some babies are born healthy and have been regarded by doctors to live a long, normal, and healthy life. Others, unfortunately, aren\u2019t so lucky. Many have to live with a disease that may cut their lives short and reduce the quality of life.<\/p>\n

This is why doctors have been working hard to find cure for all sorts of ailments. With research, dedication, and a lot of effort, many have come across help that may actually be effective when it comes to changing destiny.<\/p>\n

For the most recent case, a 19-month-old girl has become the first ever person to be cured of a rare and deadly condition called MLD. This is with the help of a new therapy that inserts functional versions of a faulty gene into their bone marrow.<\/p>\n

So, what exactly is MLD? For those who are unfamiliar with the disease, this is part of a family of illnesses<\/a>\u00a0called Lysosomal storage diseases. There are therapies such as Libmeldy available and these are thought to be potential treatments for LSDs such as Krabbe disease, X-linked adrenoleukodystrophy, Canavan disease, Zellweger disease, and many more. The diseases mentioned above are considered rare, rarely screened for at birth, typically only affects infants, and are fatal when child is over 5 to 10 years old.<\/p>\n

For the child, Teddi Shaw is now predicted to live a long, normal, healthy life. He got the said \u201ccure\u201d for metachromatic leukodystrophy (MLD).<\/p>\n

\u201cTeddi is doing absolutely brilliant,\u201d<\/strong><\/em> said the mom Ally\u00a0to the Guardian.<\/a>\u00a0\u201cShe is walking, running, a chatterbox\u2014absolutely no signs so far of MLD. She is an absolute character and has everyone around her laughing all the time.\u201d<\/strong><\/em><\/p>\n

The cure that is being discussed is also the most expensive drug in the world. As mentioned above, Libmeldy is the drug. Its main function is to correct the copies of faulty genes that cause MLD after a patient\u2019s stem cells are taken out. These cells are treated with Libmeldy before these are replaced within the bone marrow.<\/p>\n

\u201cThis is a huge moment of hope for parents and their babies born with this devastating inherited disorder, that can now be treated with a single round of revolutionary treatment,\u201d<\/strong><\/em> Amanda Pritchard\u00a0said<\/a>, she is the chief executive of NHS England,<\/p>\n

Previous data on the drug shows that as far as 8 years post-treatment, it has been able to successfully correct the underlying genetic problems completely and in its entirety.<\/p>\n

However, the drug is priced at $3 million, which is way above almost everyone\u2019s pay grade. However, the NHS agreed with Orchard Therapeutics to substantially lower the price just for Teddi.<\/p>\n

Orchard CEO Bobby Gaspar\u00a0has said<\/a>\u00a0in a statement that the price represents the costs that were needed to develop, test, and get the drug approved. This is also considering the fact that there may only be 7 to 8 diagnoses in England each year. It\u2019s also just a little less than the 10-year cost of the standard of care for patients suffering from MLD and who gradually lose motor function and control of their senses.<\/p>\n

Rates of MLD will differ between North American and European populations. They say that there may be be between 1 in 40,000 live births to 1 in 150,000 diagnosed, but as of now, many of these patients can look forward to the fact that a cure is in the horizon for them.<\/p>\n

\n","protected":false},"excerpt":{"rendered":"

Some babies are born healthy and have been regarded by doctors to live a long, normal, and healthy life. Others, unfortunately, aren\u2019t so lucky. Many have to live with a disease that may cut their lives short and reduce the quality of life. This is why doctors have been working hard to find cure for […]<\/p>\n","protected":false},"author":9,"featured_media":5315,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"image","meta":[],"categories":[4],"tags":[16,1573,1572],"yoast_head":"\nChild Diagnosed With Deadly MLD Found To Be Cured For The First Time In History<\/title>\n<meta name=\"description\" content=\"A 19-month-old girl has become the first ever person to be cured of a rare and deadly condition called MLD.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/healthnewscentral.com\/child-diagnosed-with-deadly-mld-found-to-be-cured-for-the-first-time-in-history\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Child Diagnosed With Deadly MLD Found To Be Cured For The First 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