{"id":419847,"date":"2021-01-24T09:03:18","date_gmt":"2021-01-24T14:03:18","guid":{"rendered":"http:\/\/www.marketnewsdesk.com\/?p=419847"},"modified":"2021-01-24T09:03:18","modified_gmt":"2021-01-24T14:03:18","slug":"mirum-pharmaceuticals-honors-alagille-syndromeawareness-day-with-launch-of-disease-awareness-campaign","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/mirum-pharmaceuticals-honors-alagille-syndromeawareness-day-with-launch-of-disease-awareness-campaign\/","title":{"rendered":"Mirum Pharmaceuticals Honors Alagille SyndromeAwareness Day with Launch of Disease Awareness Campaign"},"content":{"rendered":"

<\/p>\n

Mirum Pharmaceuticals Honors Alagille Syndrome<\/b>Awareness Day with Launch of Disease Awareness Campaign<\/b><\/p>\n

\n– The national campaign seeks to emphasize the unbearable burden ALGS has on patients and their families.<\/i><\/p>\n

\n– Mirum underscores its commitment to continued understanding of the rare liver disease through research and support of ALGS community.<\/i><\/p>\n

FOSTER CITY, Calif.–(BUSINESS WIRE<\/a>)–
\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and diagnose patients with the goal to improve time to diagnosis and treatment.\n<\/p>\n

\nChildren with ALGS live with severe and unrelenting symptoms that can impact their overall quality of life. Identifying these symptoms early on can lead to a diagnosis and an effective treatment plan.\n<\/p>\n

\n\u201cMany patients live for months or years before being diagnosed and some patients are misdiagnosed, leading to unnecessary and invasive surgeries that do not address the underlying cause of their liver disease,\u201d said Chris Peetz, president and chief executive officer at Mirum. \u201cOur objective is to provide a platform that helps healthcare providers understand the specific signs and symptoms of ALGS so they can be better equipped to identify and diagnose this rare disease and help their patients get the treatment they need.\u201d\n<\/p>\n

\n\u201cThis disease awareness campaign has the potential to help healthcare providers recognize Alagille syndrome in their patients in order to properly address this rare disease,\u201d said Roberta Smith, president of the Alagille syndrome Alliance and parent of a child with ALGS. \u201cThe impacts of Alagille syndrome on families are many. We have only begun to scratch the surface in understanding disease burden and specifically, how different regions in the world are affected differently. As we celebrate International Alagille Syndrome Awareness Day, we will focus on sharing Alagille syndrome family stories that emphasize the critical need for continued research and treatment options for patients worldwide. The patient voice is the Alagille syndrome community\u2019s greatest vehicle for change.\u201d\n<\/p>\n

\nMirum\u2019s goal is to advance the understanding and diagnosis of ALGS so that patients and their families will get the medical care and support they need to improve their quality of life. The company and its founders have been dedicated to understanding ALGS through the support of resources like the Global Alagille Alliance (GALA) for continued research, as well as genetic testing platforms that help effectively identify ALGS in patients. These resources assist in advancing research and the healthcare community\u2019s understanding of this devastating rare disease.\n<\/p>\n

\nVisit the Unbearable ALGS website<\/a> to learn more about Alagille syndrome and the significant impact it has on patients and their families.\n<\/p>\n

Alagille syndrome Awareness Day \u2013 January 24, 2021<\/b><\/p>\n

\nMirum is also proud to champion the Alagille syndrome Alliance and their ongoing work to support ALGS families through education, outreach, and community. This year, in celebration of International ALGS Awareness Day, they are hosting a 24-hour Aware-a-Thon<\/a> that will include family stories, messages, expert talks, and educational resources aimed at increasing the public\u2019s awareness of ALGS, a crucial element in their mission. To learn more about how to get involved and support the ALGS Alliance and the ALGS community, visit their Awareness Day website<\/a>.\n<\/p>\n

About Alagille syndrome<\/b><\/p>\n

\nAlagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people.1<\/sup> In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system.2<\/sup> The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports, 60% to 75% of patients with ALGS have a liver transplant before reaching adulthood.3<\/sup> Signs and symptoms arising from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin), and pruritus (itch)2<\/sup>. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.4<\/sup><\/p>\n

About Mirum<\/b><\/p>\n

\nMirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirum\u2019s lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has initiated a rolling NDA submission for maralixibat in the treatment of cholestatic pruritus in patients with ALGS and expects to complete the submission in the first quarter of 2021. Additionally, Mirum\u2019s marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency.\n<\/p>\n

\nMirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com<\/a>.\n<\/p>\n

\nFollow Mirum on Twitter<\/a>, Facebook<\/a>, LinkedIn<\/a> and Instagram<\/a>.\n<\/p>\n

Forward Looking Statements <\/b><\/p>\n

\nStatements contained in this press release regarding matters that are not historical facts are \u201cforward-looking statements\u201d within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the regulatory approval path for maralixibat in the United States and the European Union, and Mirum\u2019s ALGS disease awareness campaign. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as \u201cplans,\u201d \u201cgoal\u201d, \u201cwill,\u201d \u201dmay,\u201d \u201cexpects,\u201d \u201cpotential\u201d and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum\u2019s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum\u2019s business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum\u2019s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management\u2019s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.\n<\/p>\n

References<\/b><\/p>\n

1<\/sup>Danks, et al. Archives of Disease in Childhood1977
\n
2<\/sup>Johns Hopkins Medicine. hopkinsmedicine.org\/health\/conditions-and-diseases\/Alagille-syndrome
\n<\/a>
3<\/sup>Vandriel, et al. GALA EASL 2020; Kamath, et al. Hepatology Communications 2020
\n
4<\/sup>Elisofon, et al. Journal of Pediatric Gastroenterology and Nutrition 2010\n<\/p>\n

\"\"<\/p>\n

View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20210124005076\/en\/<\/a><\/span><\/p>\n

Mirum Contacts:
\n<\/b>
Media
\n
Erin Murphy
\n
media@mirumpharma.com <\/a><\/p>\n

\nInvestors
\n
Ian Clements, Ph.D.
\n
investors@mirumpharma.com<\/a><\/p>\n

KEYWORDS:<\/b> California United States North America<\/p>\n

INDUSTRY KEYWORDS:<\/b> Infectious Diseases Health Science Pharmaceutical Research<\/p>\n

MEDIA:<\/b><\/p>\n\n\n\n\n
Logo<\/b><\/font><\/td>\n<\/tr>\n
\"Logo\"<\/td>\n<\/tr>\n
<\/font><\/td>\n<\/tr>\n<\/table>\n","protected":false},"excerpt":{"rendered":"

Mirum Pharmaceuticals Honors Alagille SyndromeAwareness Day with Launch of Disease Awareness Campaign – The national campaign seeks to emphasize the unbearable burden ALGS has on patients and their families. – Mirum underscores its commitment to continued understanding of the rare liver disease through research and support of ALGS community. FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and diagnose patients with the goal to improve time to diagnosis and treatment. Children with ALGS live with … <\/p>\n

Continue reading “Mirum Pharmaceuticals Honors Alagille SyndromeAwareness Day with Launch of Disease Awareness Campaign”<\/span><\/a><\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[],"tags":[],"class_list":["post-419847","post","type-post","status-publish","format-standard","hentry"],"yoast_head":"\nMirum Pharmaceuticals Honors Alagille SyndromeAwareness Day with Launch of Disease Awareness Campaign - Market Newsdesk<\/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:\/\/www.marketnewsdesk.com\/index.php\/mirum-pharmaceuticals-honors-alagille-syndromeawareness-day-with-launch-of-disease-awareness-campaign\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Mirum Pharmaceuticals Honors Alagille SyndromeAwareness Day with Launch of Disease Awareness Campaign - Market Newsdesk\" \/>\n<meta property=\"og:description\" content=\"Mirum Pharmaceuticals Honors Alagille SyndromeAwareness Day with Launch of Disease Awareness Campaign – The national campaign seeks to emphasize the unbearable burden ALGS has on patients and their families. – Mirum underscores its commitment to continued understanding of the rare liver disease through research and support of ALGS community. FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and diagnose patients with the goal to improve time to diagnosis and treatment. 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FOSTER CITY, Calif.–(BUSINESS WIRE)– Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and diagnose patients with the goal to improve time to diagnosis and treatment. 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