Zevra Niemann-Pick disease type C drug Miplyffa now available

seekingalpha06:29

Jose Luis Pelaez Inc/DigitalVision via Getty Images Miplyffa (arimoclomol), the first ever medication specifically approved for Niemann-Pick disease type C, is now available from Zevra Therapeutics (...

Source Link

Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.

Comments

We need your insight to fill this gap

Leave a comment

{"i18n":{"language":"en_US"},"isChannel":false,"data":{"share":"https://ttm.financial/m/news/2485240344?lang=en_US&edition=fundamental","thumbnail":"https://static.seekingalpha.com/cdn/s3/uploads/getty_images/56970863/image_56970863.jpg?io=getty-c-w750","is_english":true,"pubTime":"2024-11-22 06:29","share_image_url":"https://static.laohu8.com/e9f99090a1c2ed51c021029395664489","id":"2485240344","market":"nz","top_or_hot":-1,"title":"Zevra Niemann-Pick disease type C drug Miplyffa now available","media":"seekingalpha","content":"<div>\n<p>Jose Luis Pelaez Inc/DigitalVision via Getty Images Miplyffa (arimoclomol), the first ever medication specifically approved for Niemann-Pick disease type C, is now available from Zevra Therapeutics (...</p>\n\n<a href=\"https://seekingalpha.com/news/4319157-zevra-niemann-pick-disease-type-c-drug-miplyffa-now-available\">Source Link</a>\n\n</div>\n","source":"seekingalpha_trending_news","html":"<!DOCTYPE html>\n<html>\n<head>\n<meta http-equiv=\"Content-Type\" content=\"text/html; charset=utf-8\" />\n<meta name=\"viewport\" content=\"width=device-width,initial-scale=1.0,minimum-scale=1.0,maximum-scale=1.0,user-scalable=no\"/>\n<meta name=\"format-detection\" content=\"telephone=no,email=no,address=no\" />\n<title>Zevra Niemann-Pick disease type C drug Miplyffa now available</title>\n<style type=\"text/css\">\na,abbr,acronym,address,applet,article,aside,audio,b,big,blockquote,body,canvas,caption,center,cite,code,dd,del,details,dfn,div,dl,dt,\nem,embed,fieldset,figcaption,figure,footer,form,h1,h2,h3,h4,h5,h6,header,hgroup,html,i,iframe,img,ins,kbd,label,legend,li,mark,menu,nav,\nobject,ol,output,p,pre,q,ruby,s,samp,section,small,span,strike,strong,sub,summary,sup,table,tbody,td,tfoot,th,thead,time,tr,tt,u,ul,var,video{ font:inherit;margin:0;padding:0;vertical-align:baseline;border:0 }\nbody{ font-size:16px; line-height:1.5; color:#999; background:transparent; }\n.wrapper{ overflow:hidden;word-break:break-all;padding:10px; }\nh1,h2{ font-weight:normal; line-height:1.35; margin-bottom:.6em; }\nh3,h4,h5,h6{ line-height:1.35; margin-bottom:1em; }\nh1{ font-size:24px; }\nh2{ font-size:20px; }\nh3{ font-size:18px; }\nh4{ font-size:16px; }\nh5{ font-size:14px; }\nh6{ font-size:12px; }\np,ul,ol,blockquote,dl,table{ margin:1.2em 0; }\nul,ol{ margin-left:2em; }\nul{ list-style:disc; }\nol{ list-style:decimal; }\nli,li p{ margin:10px 0;}\nimg{ max-width:100%;display:block;margin:0 auto 1em; }\nblockquote{ color:#B5B2B1; border-left:3px solid #aaa; padding:1em; }\nstrong,b{font-weight:bold;}\nem,i{font-style:italic;}\ntable{ width:100%;border-collapse:collapse;border-spacing:1px;margin:1em 0;font-size:.9em; }\nth,td{ padding:5px;text-align:left;border:1px solid #aaa; }\nth{ font-weight:bold;background:#5d5d5d; }\n.symbol-link{font-weight:bold;}\n/* header{ border-bottom:1px solid #494756; } */\n.title{ margin:0 0 8px;line-height:1.3;color:#ddd; }\n.meta {color:#5e5c6d;font-size:13px;margin:0 0 .5em; }\na{text-decoration:none; color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 11px; color: #7E829C; margin: 0;line-height: 11px;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nZevra Niemann-Pick disease type C drug Miplyffa now available\n</h2>\n\n<h4 class=\"meta\">\n\n\n2024-11-22 06:29 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://seekingalpha.com/news/4319157-zevra-niemann-pick-disease-type-c-drug-miplyffa-now-available><strong>seekingalpha</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>Jose Luis Pelaez Inc/DigitalVision via Getty Images Miplyffa (arimoclomol), the first ever medication specifically approved for Niemann-Pick disease type C, is now available from Zevra Therapeutics (...</p>\n\n<a href=\"https://seekingalpha.com/news/4319157-zevra-niemann-pick-disease-type-c-drug-miplyffa-now-available\">Source Link</a>\n\n</div>\n\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"LU1221951129.SGD","symbol_name":"NORDEA 1 STABLE RETURN \"HM\" (SGDHDG) INC","start_time":0,"source_url":"https://seekingalpha.com/news/4319157-zevra-niemann-pick-disease-type-c-drug-miplyffa-now-available","article_id":"2485240344","we_media_id":null,"thumbnails":["https://static.seekingalpha.com/cdn/s3/uploads/getty_images/56970863/image_56970863.jpg?io=getty-c-w750"],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2485240344","pubTimestamp":1732228187,"sourceInfo":{"source_id":"seekingalpha_trending_news","name":"Seeking Alpha Trending News"},"weMediaInfo":null,"summary":"Jose Luis Pelaez Inc/DigitalVision via Getty Images Miplyffa , the first ever medication specifically approved for Niemann-Pick disease type C, is now available from Zevra Therapeutics . The drug is approved for use in combination with Johnson & Johnson's  Zavesca  for the treatment of patients two years and older. Zavesca is approved for Gaucher disease.  Miplyffa works by boosting the activation of the transcription factors EB  and E3 , leading to the upregulation of coordinated lysosomal expression and regulation  genes. The U.S. FDA approved Miplyffa in September.","collect":0,"end_time":0,"defaultTopTitle":"seekingalpha.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"LU1221951129.SGD":"NORDEA 1 STABLE RETURN \"HM\" (SGDHDG) INC","BK4007":"制药","SG9999002224.SGD":"Allianz Global High Payout SGD","LU1894683264.USD":"AMUNDI FUNDS US EQUITY RESEARCH VALUE \"A2\" (USD) ACC","LU1221951046.USD":"NORDEA 1 STABLE RETURN \"HM\" (USDHDG) INC","LU0345770308.USD":"NINETY ONE GSF GLOBAL STRATEGIC EQUITY \"A\" (USD) ACC","LU1066051498.USD":"HSBC GIF GLOBAL EQUITY VOLATILITY FOCUSED \"AM2\" (USD) INC","BK4559":"巴菲特持仓","LU0882574055.USD":"富达全球健康医疗A ACC","LU1066053197.SGD":"HSBC GIF GLOBAL EQUITY VOLATILITY FOCUSED \"AM3\" (SGDHDG) INC","LU1032466523.USD":"高盛全球多资产收益组合Acc","BK4550":"红杉资本持仓","BK4568":"美国抗疫概念","LU1674673691.USD":"HSBC GIF GLOBAL LOWER CARBON EQUITY \"AD\" (USD) INC","IE0002270589.USD":"LEGG MASON CLEARBRIDGE VALUE \"A\" (USD) INC","IE00BGHQF631.EUR":"GUINNESS GLOBAL EQUITY INCOME \"C\" (EUR) ACC","LU1674673428.USD":"HSBC GIF GLOBAL LOWER CARBON EQUITY \"AC\" (USD) ACC","IE0002141913.USD":"JANUS HENDERSON GLOBAL LIFE SCIENCES \"I2\" (USD) ACC","IE00B3PB1722.GBP":"GUINNESS GLOBAL EQUITY INCOME \"C\" (GBP) INC","IE00B42XCP33.USD":"GUINNESS GLOBAL EQUITY INCOME \"C\" (USD) INC","LU2133065610.SGD":"JPMorgan Investment Funds - Global Dividend A (mth) SGD","LU0234572021.USD":"高盛美国核心股票组合Acc","IE00BFTCPJ56.SGD":"Janus Henderson Global Life Sciences A Acc SGD","ZVRA":"Zevra Therapeutics","LU1261432733.SGD":"Fidelity World A-ACC-SGD","IE00B4R5TH58.HKD":"JANUS HENDERSON GLOBAL LIFE SCIENCES \"A2\" (HKD) ACC","LU1732799900.SGD":"JPMorgan Investment Funds - Global Income A (irc) SGD-H","LU1066051225.USD":"HSBC GIF GLOBAL EQUITY VOLATILITY FOCUSED \"AC\" (USD) ACC","LU0889566641.SGD":"FTSF - Templeton Shariah Global Equity A Acc SGD","IE00B7SZLL34.SGD":"Legg Mason ClearBridge - Value A Acc SGD-H","LU0234570918.USD":"高盛全球核心股票组合Acc Close","IE00BSNM7G36.USD":"NEUBERGER BERMAN SYSTEMATIC GLOBAL SUSTAINABLE VALUE \"A\" (USD) ACC","LU1935042991.SGD":"MANULIFE GF GLOBAL MULTI-ASSET DIVERSIFIED INCOME  \"AA\" (SGDHDG) INC","LU0345769128.USD":"NINETY ONE GSF GLOBAL EQUITY \"A\" (USD) ACC","LU0792757196.USD":"TEMPLETON SHARIAH GLOBAL EQUITY FUND \"A\" (USD) ACC","LU1506573853.SGD":"MANULIFE GF GLOBAL EQUITY \"AA\" (SGD) INC","IE00BVYPNW00.USD":"GUINNESS GLOBAL EQUITY INCOME \"C\" (USD) ACC","LU1066051811.HKD":"HSBC GIF GLOBAL EQUITY VOLATILITY FOCUSED \"AM2\" (HKD) INC","LU1023059063.AUD":"BGF WORLD HEALTHSCIENCE \"A2\" (AUDHDG) ACC","BK4532":"文艺复兴科技持仓","LU1732800096.USD":"摩根大通环球收益基金A (irc)","LU0289739699.SGD":"AB INTERNATIONAL HEALTH CARE PORTFOLIO \"A\" (SGD) ACC","IE00B19Z3B42.SGD":"Legg Mason ClearBridge - Value A Acc SGD","JNJ":"强生","LU1057294990.SGD":"Blackrock World Healthscience A2 SGD-H","LU0640476718.USD":"THREADNEEDLE (LUX) US CONTRARIAN CORE EQ \"AU\" (USD) ACC","IE00BJT1NW94.SGD":"JANUS HENDERSON GLOBAL LIFE SCIENCES \"A2\" (SGDHDG) ACC","BK4534":"瑞士信贷持仓","LU1280957306.USD":"THREADNEEDLE (LUX) US CONTRARIAN CORE EQUITIES \"AUP\" (USD) INC","LU1914381329.SGD":"Allianz Best Styles Global Equity Cl ET Acc H2-SGD"},"translate_title":"Zevra尼曼-皮克病C型药物Miplyffa上市","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"JNJ":1,"ZVRA":0.9},"content_text":"Jose Luis Pelaez Inc/DigitalVision via Getty Images Miplyffa (arimoclomol), the first ever medication specifically approved for Niemann-Pick disease type C, is now available from Zevra Therapeutics (NASDAQ:ZVRA). The drug is approved for use in combination with Johnson & Johnson's (JNJ) Zavesca (miglustat) for the treatment of patients two years and older. Zavesca is approved for Gaucher disease.  Miplyffa works by boosting the activation of the transcription factors EB (TFEB) and E3 (TFE3), leading to the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. The U.S. FDA approved Miplyffa in September.","kind":null,"is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"Product Release","news_rank":0,"symbols":[],"gpt_button":1},"commentList":[],"isCommentEnd":true,"newsSizeData":{"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"likeStatus":false,"favoriteStatus":false},"APP":{"userAgent":"Mozilla/5.0 AppleWebKit/537.36 (KHTML, like Gecko; compatible; ClaudeBot/1.0; +claudebot@anthropic.com)","isDev":false,"isTTM":true,"deviceId":"web-server-community-laohu8-v3","version":"4.29.2","shortVersion":"4.29.2","platform":"web","vendor":"web","appName":"ttm","isIOS":false,"isAndroid":false,"isTiger":false,"isTHS":false,"isWeiXin":false,"isWeiXinMini":false,"isWeiBo":false,"isQQ":false,"isBaiduSwan":false,"isBaiduBox":false,"isDingTalk":false,"isToutiao":false,"isOnePlus":false,"isHuaWei":false,"isXiaomi":false,"isXiaomiWebView":false,"isOppo":false,"isVivo":false,"isSamsung":false,"isMobile":false},"href":"/m/news/2485240344"}