{"id":1630,"date":"2017-11-30T20:08:04","date_gmt":"2017-11-30T20:08:04","guid":{"rendered":"https:\/\/www.mybiosource.com\/learn\/?p=1630"},"modified":"2023-03-06T17:49:16","modified_gmt":"2023-03-06T17:49:16","slug":"fda-approves-mepsevii-treat-sly-syndrome","status":"publish","type":"post","link":"https:\/\/www.mybiosource.com\/learn\/fda-approves-mepsevii-treat-sly-syndrome\/","title":{"rendered":"FDA approves Mepsevii to treat Sly Syndrome"},"content":{"rendered":"

Table of Contents<\/strong><\/span><\/h2>\n

1. Introduction<\/strong>
\n2. What is Sly Syndrome?<\/strong>
\n3. Symptoms of Sly Syndrome<\/strong>
\n4. Current treatment options <\/strong>
\n5. Mepsevii: The First FDA-Approved Treatment for Sly Syndrome<\/strong>
\n6. How Mepsevii Works<\/strong>
\n7. Safety and Efficacy of Mepsevii<\/strong>
\n8. Treatment Duration for Mepsevii<\/strong>
\n9. Conclusion<\/strong><\/p>\n

\n

The U.S. Food and Drug Administration<\/span> (FDA) approved Mepsevii to treat pediatric and adult patients with the genetic metabolic condition called mucopolysaccharidosis<\/span> type VII. Incidentally, this is the first FDA approved treatment option available for people with Sly Syndrome. <\/em>
\nThe symptoms of the Sly syndrome (also known as mucopolysaccharidosis<\/span> type VII) are short stature due to growth retardation, bone weakness, and mental illness. In milder cases, osteoarthritis is a common complication. Around 150 patients across the world had been diagnosed with Sly Syndrome. <\/em>
\nCaused by a deficiency of the enzyme beta-glucuronidase, the Sly syndrome is characterized by an accumulation of dermatan sulfate<\/span> DS), heparan sulfate<\/span> (HS) and chondroitin sulfate<\/span> (CS) in tissues and organs of the body including the central nervous system<\/span>. The symptoms, in some cases, also include abnormalities of the intestines, corneas of the eyes and skeletal system. In some patients, valve abnormalities, enlarged liver and spleen<\/span>, and narrowed airways are also observed leading to lung infections and breathing problems.<\/em>
\nThe standard treatment for Sly syndrome includes surgical correction of bones, hernias, Ocular and cardiovascular<\/span> abnormalities. In recent times. stem cell<\/span> therapies have also shown promise in treating Sly Syndrome. The life expectancy of individuals with MPS VII depends on the severity of symptoms and Some affected individuals do not survive infancy, while others may live into adolescence or adulthood. Heart disease<\/span> and airway obstruction are major causes of death in people with MPS VII.<\/em>
\nThe approved drug, Mepsevii, replaces the missing enzyme, beta-glucuronidase, therefore inhibiting the accumulation of toxic materials in the body\u2019s cells. The safety and efficacy were established in individuals with MPS VII of age range 5 \u2013 25 years. The treatment duration is up to 164 weeks which involves intake of the drug at doses of up to 4 mg\/kg once every two weeks.<\/em><\/p>\n

References<\/strong><\/em>
\nhttps:\/\/rarediseases.org\/rare-diseases\/sly-syndrome\/<\/em><\/p>\n

https:\/\/www.fda.gov\/NewsEvents\/Newsroom\/PressAnnouncements\/ucm585308.htm<\/em><\/p>\n

 <\/p>\n

 <\/p>\n","protected":false},"excerpt":{"rendered":"

Table of Contents 1. Introduction 2. What is Sly Syndrome? 3. Symptoms of Sly Syndrome 4. Current treatment options 5. Mepsevii: The First FDA-Approved Treatment for Sly Syndrome 6. How Mepsevii Works 7. Safety and Efficacy of Mepsevii 8. Treatment Duration for Mepsevii 9. Conclusion The U.S. Food and Drug Administration (FDA) approved Mepsevii to […]<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"categories":[1],"tags":[16],"class_list":["post-1630","post","type-post","status-publish","format-standard","hentry","category-uncategorized","tag-sly-syndrome"],"_links":{"self":[{"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/posts\/1630","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/comments?post=1630"}],"version-history":[{"count":0,"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/posts\/1630\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/media?parent=1630"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/categories?post=1630"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.mybiosource.com\/learn\/wp-json\/wp\/v2\/tags?post=1630"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}