{"id":1818,"date":"2023-01-13T15:36:31","date_gmt":"2023-01-13T15:36:31","guid":{"rendered":"https:\/\/blogs.bmj.com\/jmg\/?p=1818"},"modified":"2026-02-23T00:21:14","modified_gmt":"2026-02-23T00:21:14","slug":"long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes","status":"publish","type":"post","link":"https:\/\/blogs.bmj.com\/jmg\/2023\/01\/13\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\/","title":{"rendered":"Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes (Contributed by Prof. Derralynn Hughes)"},"content":{"rendered":"<p>Fabry disease is a rare inherited condition that can lead to serious renal, cardiac and cerebrovascular events. Using pooled clinical trial data, we examined the efficacy of migalastat, an oral treatment, in a large group of patients. The rate of newly occurring Fabry-associated clinical events (FACES) with prolonged treatment was low, regardless of whether patients had previously received enzyme replacement therapy (ERT). FACES were more common in patients who had renal impairment at study entry. Findings suggest that migalastat is an effective long-term treatment option for patients with Fabry disease. Preserving kidney function and timely treatment initiation instead of a watch-and-wait monitoring strategy may improve patient outcomes. (<a href=\"https:\/\/jmg.bmj.com\/content\/early\/2022\/12\/21\/jmg-2022-108669\">https:\/\/jmg.bmj.com\/content\/early\/2022\/12\/21\/jmg-2022-108669<\/a> )<\/p>\n<p><a href=\"https:\/\/blogs.bmj.com\/jmg\/2023\/01\/13\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\/picture1-2\/\" rel=\"attachment wp-att-1819\"><img loading=\"lazy\" decoding=\"async\" class=\"alignnone size-medium wp-image-1819\" src=\"https:\/\/blogs.bmj.com\/jmg\/files\/2023\/01\/Picture1-300x172.jpg\" alt=\"\" width=\"300\" height=\"172\" srcset=\"https:\/\/blogs.bmj.com\/jmg\/files\/2023\/01\/Picture1-300x172.jpg 300w, https:\/\/blogs.bmj.com\/jmg\/files\/2023\/01\/Picture1.jpg 624w\" sizes=\"auto, (max-width: 300px) 100vw, 300px\" \/><\/a><\/p>\n<p>eGFR, estimated glomerular filtration rate, was used to indicate renal impairment<!--TrendMD v2.4.8--><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Fabry disease is a rare inherited condition that can lead to serious renal, cardiac and cerebrovascular events. Using pooled clinical trial data, we examined the efficacy of migalastat, an oral treatment, in a large group of patients. The rate of newly occurring Fabry-associated clinical events (FACES) with prolonged treatment was low, regardless of whether patients [&#8230;]<\/p>\n<p><a class=\"btn btn-secondary understrap-read-more-link\" href=\"https:\/\/blogs.bmj.com\/jmg\/2023\/01\/13\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\/\">Read More&#8230;<\/a><\/p>\n","protected":false},"author":123,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-1818","post","type-post","status-publish","format-standard","hentry","category-uncategorized"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.6 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes (Contributed by Prof. Derralynn Hughes) - JMG Contact blog<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/blogs.bmj.com\/jmg\/2023\/01\/13\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes (Contributed by Prof. Derralynn Hughes) - JMG Contact blog\" \/>\n<meta property=\"og:description\" content=\"Fabry disease is a rare inherited condition that can lead to serious renal, cardiac and cerebrovascular events. Using pooled clinical trial data, we examined the efficacy of migalastat, an oral treatment, in a large group of patients. The rate of newly occurring Fabry-associated clinical events (FACES) with prolonged treatment was low, regardless of whether patients [...]Read More...\" \/>\n<meta property=\"og:url\" content=\"https:\/\/blogs.bmj.com\/jmg\/2023\/01\/13\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\/\" \/>\n<meta property=\"og:site_name\" content=\"JMG Contact blog\" \/>\n<meta property=\"article:published_time\" content=\"2023-01-13T15:36:31+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2026-02-23T00:21:14+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/blogs.bmj.com\/jmg\/files\/2023\/01\/Picture1.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"624\" \/>\n\t<meta property=\"og:image:height\" content=\"357\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"hqqu\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@HuiQiQu\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"hqqu\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"1 minute\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2023\\\/01\\\/13\\\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2023\\\/01\\\/13\\\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\\\/\"},\"author\":{\"name\":\"hqqu\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#\\\/schema\\\/person\\\/be0250f8d5b52412c3e7c222dabd591b\"},\"headline\":\"Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes (Contributed by Prof. Derralynn Hughes)\",\"datePublished\":\"2023-01-13T15:36:31+00:00\",\"dateModified\":\"2026-02-23T00:21:14+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2023\\\/01\\\/13\\\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\\\/\"},\"wordCount\":146,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/#organization\"},\"image\":{\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2023\\\/01\\\/13\\\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/files\\\/2023\\\/01\\\/Picture1-300x172.jpg\",\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2023\\\/01\\\/13\\\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\\\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2023\\\/01\\\/13\\\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\\\/\",\"url\":\"https:\\\/\\\/blogs.bmj.com\\\/jmg\\\/2023\\\/01\\\/13\\\/long-term-multisystemic-efficacy-of-migalastat-on-fabry-associated-clinical-events-including-renal-cardiac-and-cerebrovascular-outcomes\\\/\",\"name\":\"Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes (Contributed by Prof. Derralynn Hughes) - 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