{"id":457263,"date":"2021-03-15T07:03:11","date_gmt":"2021-03-15T11:03:11","guid":{"rendered":"http:\/\/www.marketnewsdesk.com\/?p=457263"},"modified":"2021-03-15T07:03:11","modified_gmt":"2021-03-15T11:03:11","slug":"amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\/","title":{"rendered":"Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; Scientific Conference"},"content":{"rendered":"<div class=\"mw_release\">\n<p align=\"justify\">PHILADELPHIA, March  15, 2021  (GLOBE NEWSWIRE) &#8212; <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=ZtNz4tnnx89oKZwSuNE0u3fzFzKTB4yBiG8-JF43C5L4lZz1Z_X8tU0ham928vdwtM9zwDxW-UJkumqc4LbhG9ZuYnClmUsHigo9fqsfv9U=\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">Amicus Therapeutics<\/a> (Nasdaq: FOLD), today announced one oral presentation and two posters highlighting its development program for Pompe disease will be included at the <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=TQp9k0bLY0K5qbjOt_I-rqaAYZI5yCFhPNXV5Ki7TNWOMaB8IqKGX9t9_teYb1gnZMrZ35yhEJjvoIe727zm9hZwgr4YOvBXbzR0Ti4PFnhT5cACcZNY3DnZ1PMiiJFWc__Nw4M6VyCroUbmXvONEA==\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">2021 MDA Virtual Clinical &amp; Scientific Conference<\/a> to be held March 15-18, 2021.<\/p>\n<p align=\"justify\">\n        <strong>Oral Platform Presentation: Thursday, March 18, 5:30 \u2013 5:45 p.m. ET<\/strong>\n      <\/p>\n<ul type=\"disc\">\n<li style=\"text-align:justify\">\n          <em>Efficacy and safety of AT-GAA (cipaglucosidase alfa\/miglustat) versus alglucosidase alfa\/placebo in late-onset Pompe disease (LOPD): A phase 3 trial (PROPEL)<\/em><\/p>\n<ul type=\"circle\">\n<li style=\"text-align:justify\">Presenter: Tahseen Mozaffar, MD, FAAN, University of California, Irvine, CA, USA<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p align=\"justify\">\n        <strong>Poster Presentations: <\/strong>\n      <\/p>\n<ul type=\"disc\">\n<li style=\"margin-bottom:6pt;text-align:justify\">\n          <em>Characterization of Response to Enzyme Replacement Therapy in Patients With Late-Onset Pompe Disease: A Retrospective Chart Review<\/em><\/p>\n<ul type=\"circle\">\n<li style=\"margin-bottom:6pt;text-align:justify\">Presenter: Priya Kishnani, MD, MBBS, Duke University Medical Center, Durham, NC, USA<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<ul type=\"disc\">\n<li style=\"margin-bottom:6pt;text-align:justify\">\n          <em>Enhancing Delivery of Acid Alpha-Glucosidase (GAA) to Skeletal Muscle in Pompe Disease (PD):\u202fKey Challenges and Attributes of AT-GAA<\/em><\/p>\n<ul type=\"circle\">\n<li style=\"margin-bottom:6pt;text-align:justify\">Presenter: Nithya Selvan, Ph.D., Amicus Therapeutics, Philadelphia, PA, USA<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p align=\"justify\">The posters and presentation will be made available on the Amicus <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=xse0dKUY7fTeJKaHtPUT4YEOHPMP9tg22eb_OiGtGZq__eJQry-F5CthQ6YZVkPhNijwRnvP_A0VOFBGB6iT9aIWAa7wfLsSPLFmPMODxKc=\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">website<\/a> following their respective presentations at the conference.<\/p>\n<p align=\"justify\">For more information on the 2021 MDA Virtual Clinical &amp; Scientific Conference, please visit <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=b6SeBQTmJvXVn7cA59k5Ti-83ya4EZ9RE0wQuXdnU6FIu8XwDDpEdmKqdSxhjdkZPEJd_p6Cu9HQZ1DiUXOMOi6Ep6EEzRskdeTGLu7PY48=\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">www.mdaconference.org<\/a>.<\/p>\n<p align=\"justify\">\n        <strong><br \/>\n          <u>About AT-GAA<\/u><br \/>\n        <\/strong><br \/>\n        <br \/>\n        <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=W9QRXJ6JCD302ym8BHuMNhZbLLOQsnX9wBSOCWERqQ7ismgPDSS8N0-S0FUOyHSBSIaxLRN7N3jh-7hZBRHE-6NVFhYgvMSePTjZ_ufohTVFsh9DTgyZtY7ltbJF1NVZ\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">AT-GAA<\/a>\u00a0is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength.<\/p>\n<p align=\"justify\">In addition, Amicus is enrolling an open-label, uncontrolled, multicenter study to evaluate the PK, safety, efficacy, and PD of AT-GAA in pediatric patients aged 12 to &lt;18 years with LOPD (ATB200-04). More information, including a list of participating sites, is available at\u00a0<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=0cjso-GGoDuSs3H_swiTfPyQB-qIrj_9hq_EiZUQRM5vcSiYYxoECjwjGH-QMza5cFauFO2a1xHXdt1t4x_YBeId_BkvGMj3l30kYLlOc9M=\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">www.clinicaltrials.gov<\/a>: NCT03911505<\/p>\n<p align=\"justify\">\n        <strong><br \/>\n          <u>About Pompe Disease<\/u><br \/>\n        <\/strong><br \/>\n        <br \/>\n        <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=rdcqvyp1V4YU6khSWSrX5yRvLl10zXmkBHcK9V6SgrUKOUDgx-IQ6icCbgwHOVxjzd5nimG5jPjx5Opd8HQ4iTxRTllKxb0BiOIjhRWJlqYAHo2EWQMb5gxtvd_2HtqI\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">Pompe disease<\/a>\u00a0is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.<\/p>\n<p align=\"justify\">\n        <strong><br \/>\n          <u>About\u00a0Amicus Therapeutics<\/u><br \/>\n        <\/strong><br \/>\n        <br \/>Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company\u2019s website at <a href=\"https:\/\/www.globenewswire.com\/Tracker?data=mpB1J6siz6kLl8PT--WlNUQAW2Pht4W3JsUMzRHwyJPd3mgWMQ_gkl2KWurFEDb0-SkLicc3hetohryapic0Lg==\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">www.amicusrx.com<\/a>, and follow us on\u00a0<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=qShbexd-_opatmsV8r_vQSHY3hMUGQKwHYKQltiQwAvV6q4MMZ7Ghdr1eLNb4hBIICCCBqQA9tXgn3qRP_QOGw==\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">Twitter<\/a>\u00a0and\u00a0<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=GiqIOwFc8RoCGFW1bs9dwGzv7t602uCbFj7T__92n3XJNma4F4ATm1MtdzHChZaIisQRxlW8pJ1yZW7riNoelN4Wfyy6KaMrEZBgAM2e8JvZpwSvVUxjYPW0eOLeAx8an-nDjmidcNCzAd-ZiWe1ZCPw6qqMeJPyTNePzMkJwqWyh38r4c1h--PW_FkTdVd_I8Ao_t2jkaXEW2RP6w9UZlkxeZ_ek_66nqziS-AOynf4EGWFPz5E-Ulz987RMzdHLgSPNttuVoc6GXB_XBMFkVva_XwN9ZWC0BNDPLV5K9VNA5FF6txmjyI65vNi6JaBvJN-YOpyt15pELuWdS6pp4BoWSrsp51qkCp0v_8CQOO2L-Yz8By1HGDhvyg7weaxPGVblsMkHgVo0zc8r3Duh_QXs0DDyxk3pydrC1-CvjLn41IjcHFgQQOftMZ8qf904aTKeag18eN3OHmigCsHig==\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">LinkedIn<\/a>.<\/p>\n<p align=\"justify\">CONTACTS:<\/p>\n<p>\n        <strong>Investors:<br \/><\/strong>Amicus Therapeutics<br \/>Andrew Faughnan<br \/>Sr. Director, Investor Relations<br \/><a href=\"https:\/\/www.globenewswire.com\/Tracker?data=_ei6K2GdYGVct5Zia6b9uyj4cA4YekveHkRoWfxHwVuuTqdGCXwPXY92dG4YR9Mk5x2Va0wjnwjFNQbmKbIW1GCmWBrFTC8-UKefRlbNaaA=\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">afaughnan@amicusrx.com<\/a>\u00a0 <br \/>(609) 662-3809<\/p>\n<p>\n        <strong>Media:<\/strong><br \/>\n        <br \/>Amicus Therapeutics<br \/>Diana Moore<br \/>Head of Global Corporate Communications<br \/><a href=\"https:\/\/www.globenewswire.com\/Tracker?data=zmzUgTQNI1CWsY-uv0eGrbl1qrIQfYE8yJfKQ4M0roaTPdck6UQsr1xRZ8U7D9InoLu7Nq6xax5zsqAzWDGOdf3nb-gsDWmLPqmGNKbVO9M=\" rel=\"nofollow noopener noreferrer\" target=\"_blank\">dmoore@amicusrx.com<\/a><a href=\"https:\/\/www.globenewswire.com\/Tracker?data=hO3-L3PmTahuyYU3p0YxKk-y0aZNF5VxY8o2pcWXfl3pPvaefOtl5q0SXtcljPd7MRsEsWKBAUNpco1umzwHm24aoz03vMiAS8j0nIdGVKvs6Q_1ymO9jcAMJOrRQV87AsDpnTB-IHWTbiN3Te5r4ZsK6Jw-dCm6gfmFpJR6eKROtY1FoFQ4sFW4nUlV0E06XrgLn4-HESb2qV1L6ar2NA==\" rel=\"nofollow noopener noreferrer\" target=\"_blank\"><br \/><\/a>(609) 662-5079<\/p>\n<p align=\"justify\">FOLD\u2013G<\/p>\n<p>      <img loading=\"lazy\" decoding=\"async\" class=\"__GNW8366DE3E__IMG\" src=\"https:\/\/www.globenewswire.com\/newsroom\/ti?nf=ODE4OTcwNyM0MDYzMjI4IzIwMDU2NTg=\" width=\"1\" height=\"1\" \/><br \/>\n      <br \/>\n      <img loading=\"lazy\" decoding=\"async\" class=\"__GNW8366DE3E__IMG\" src=\"https:\/\/ml.globenewswire.com\/release\/track\/0e94fa7f-5e31-45a5-980e-45afb9a6269a\" width=\"1\" height=\"1\" \/>\n    <\/div>\n<div class=\"mw_contactinfo\"><\/div>\n","protected":false},"excerpt":{"rendered":"<p>PHILADELPHIA, March 15, 2021 (GLOBE NEWSWIRE) &#8212; Amicus Therapeutics (Nasdaq: FOLD), today announced one oral presentation and two posters highlighting its development program for Pompe disease will be included at the 2021 MDA Virtual Clinical &amp; Scientific Conference to be held March 15-18, 2021. Oral Platform Presentation: Thursday, March 18, 5:30 \u2013 5:45 p.m. ET Efficacy and safety of AT-GAA (cipaglucosidase alfa\/miglustat) versus alglucosidase alfa\/placebo in late-onset Pompe disease (LOPD): A phase 3 trial (PROPEL) Presenter: Tahseen Mozaffar, MD, FAAN, University of California, Irvine, CA, USA Poster Presentations: Characterization of Response to Enzyme Replacement Therapy in Patients With Late-Onset Pompe Disease: A Retrospective Chart Review Presenter: Priya Kishnani, MD, MBBS, Duke University Medical Center, Durham, NC, USA Enhancing Delivery of &hellip; <\/p>\n<p class=\"link-more\"><a href=\"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\/\" class=\"more-link\">Continue reading<span class=\"screen-reader-text\"> &#8220;Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; Scientific Conference&#8221;<\/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-457263","post","type-post","status-publish","format-standard","hentry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.5 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; Scientific Conference - 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\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; Scientific Conference - Market Newsdesk\" \/>\n<meta property=\"og:description\" content=\"PHILADELPHIA, March 15, 2021 (GLOBE NEWSWIRE) &#8212; Amicus Therapeutics (Nasdaq: FOLD), today announced one oral presentation and two posters highlighting its development program for Pompe disease will be included at the 2021 MDA Virtual Clinical &amp; Scientific Conference to be held March 15-18, 2021. Oral Platform Presentation: Thursday, March 18, 5:30 \u2013 5:45 p.m. ET Efficacy and safety of AT-GAA (cipaglucosidase alfa\/miglustat) versus alglucosidase alfa\/placebo in late-onset Pompe disease (LOPD): A phase 3 trial (PROPEL) Presenter: Tahseen Mozaffar, MD, FAAN, University of California, Irvine, CA, USA Poster Presentations: Characterization of Response to Enzyme Replacement Therapy in Patients With Late-Onset Pompe Disease: A Retrospective Chart Review Presenter: Priya Kishnani, MD, MBBS, Duke University Medical Center, Durham, NC, USA Enhancing Delivery of &hellip; Continue reading &quot;Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; Scientific Conference&quot;\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\/\" \/>\n<meta property=\"og:site_name\" content=\"Market Newsdesk\" \/>\n<meta property=\"article:published_time\" content=\"2021-03-15T11:03:11+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.globenewswire.com\/newsroom\/ti?nf=ODE4OTcwNyM0MDYzMjI4IzIwMDU2NTg=\" \/>\n<meta name=\"author\" content=\"Newsdesk\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Newsdesk\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/index.php\\\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\\\/\"},\"author\":{\"name\":\"Newsdesk\",\"@id\":\"https:\\\/\\\/www.marketnewsdesk.com\\\/#\\\/schema\\\/person\\\/482f27a394d4fda80ecb5499e519d979\"},\"headline\":\"Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; 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Oral Platform Presentation: Thursday, March 18, 5:30 \u2013 5:45 p.m. ET Efficacy and safety of AT-GAA (cipaglucosidase alfa\/miglustat) versus alglucosidase alfa\/placebo in late-onset Pompe disease (LOPD): A phase 3 trial (PROPEL) Presenter: Tahseen Mozaffar, MD, FAAN, University of California, Irvine, CA, USA Poster Presentations: Characterization of Response to Enzyme Replacement Therapy in Patients With Late-Onset Pompe Disease: A Retrospective Chart Review Presenter: Priya Kishnani, MD, MBBS, Duke University Medical Center, Durham, NC, USA Enhancing Delivery of &hellip; Continue reading \"Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; Scientific Conference\"","og_url":"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\/","og_site_name":"Market Newsdesk","article_published_time":"2021-03-15T11:03:11+00:00","og_image":[{"url":"https:\/\/www.globenewswire.com\/newsroom\/ti?nf=ODE4OTcwNyM0MDYzMjI4IzIwMDU2NTg=","type":"","width":"","height":""}],"author":"Newsdesk","twitter_card":"summary_large_image","twitter_misc":{"Written by":"Newsdesk","Est. reading time":"2 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\/#article","isPartOf":{"@id":"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-presentation-and-posters-at-the-2021-mda-clinical-scientific-conference\/"},"author":{"name":"Newsdesk","@id":"https:\/\/www.marketnewsdesk.com\/#\/schema\/person\/482f27a394d4fda80ecb5499e519d979"},"headline":"Amicus Therapeutics Announces Presentation and Posters at the 2021 MDA Clinical &amp; 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