{"id":5051,"date":"2022-07-06T14:48:34","date_gmt":"2022-07-06T12:48:34","guid":{"rendered":"https:\/\/www.alpha1-deutschland.org\/?p=5051"},"modified":"2022-07-06T14:48:34","modified_gmt":"2022-07-06T12:48:34","slug":"der-lange-weg-zu-alpha-1-medikamenten","status":"publish","type":"post","link":"https:\/\/alpha1-deutschland.org\/en\/der-lange-weg-zu-alpha-1-medikamenten","title":{"rendered":"The long road to Alpha-1 drugs"},"content":{"rendered":"<div id=\"fws_6aa855ad6ad8c\"  data-column-margin=\"default\" data-midnight=\"dark\"  class=\"wpb_row vc_row-fluid vc_row full-width-section\"  style=\"padding-top: 0px; padding-bottom: 0px; \"><div class=\"row-bg-wrap\" data-bg-animation=\"none\" data-bg-animation-delay=\"\" data-bg-overlay=\"false\"><div class=\"inner-wrap row-bg-layer\" ><div class=\"row-bg viewport-desktop\"  style=\"\"><\/div><\/div><\/div><div class=\"row_col_wrap_12 col span_12 dark left\">\n\t<div  class=\"vc_col-sm-12 wpb_column column_container vc_column_container col no-extra-padding inherit_tablet inherit_phone\"  data-padding-pos=\"all\" data-has-bg-color=\"false\" data-bg-color=\"\" data-bg-opacity=\"1\" data-animation=\"\" data-delay=\"0\" >\n\t\t<div class=\"vc_column-inner\" >\n\t\t\t<div class=\"wpb_wrapper\">\n\t\t\t\t<div class=\"vc_separator wpb_content_element vc_separator_align_center vc_sep_width_100 vc_sep_border_width_5 vc_sep_pos_align_center vc_sep_color_grey wpb_content_element vc_separator-has-text\" ><span class=\"vc_sep_holder vc_sep_holder_l\"><span class=\"vc_sep_line\"><\/span><\/span><h4>Author<\/h4><span class=\"vc_sep_holder vc_sep_holder_r\"><span class=\"vc_sep_line\"><\/span><\/span>\n<\/div>\n<div class=\"wpb_text_column wpb_content_element\" >\n\t<p style=\"font-weight: 400;\">Malin Fromme and Pavel Strnad<\/p>\n<\/div>\n\n\n\n<div class=\"divider-wrap\" data-alignment=\"default\"><div style=\"margin-top: 25px; height: 5px; margin-bottom: 25px;\" data-width=\"100%\" data-animate=\"\" data-animation-delay=\"\" data-color=\"default\" class=\"divider-border\"><\/div><\/div>\n<div class=\"wpb_text_column wpb_content_element\" >\n\t<p>We are frequently asked why there are so few approved medications for patients with alpha-1 antitrypsin deficiency and why, for example, augmentation therapy (replacement therapy) is not covered by health insurance in all European countries. To answer these questions, one must first understand the requirements that the relevant regulatory authorities place on potential medications.<\/p>\n<p>The most important questions are:<\/p>\n<ul>\n<li>Is the medication even effective?<\/li>\n<li>Which patients will benefit from the new drug?<\/li>\n<\/ul>\n<p>Regarding efficacy, so-called hard endpoints are usually expected. This means that a drug should be able to save lives. This hurdle is particularly difficult to overcome in the case of rare diseases that progress very slowly. The best approach is a good understanding of disease progression. It is therefore important to understand how and in whom the disease progresses and how the progression is characterized. With the help of this information, it is possible to define which parameters predict an unfavorable prognosis. This kills two birds with one stone. On the one hand, it can be defined which subjects are particularly susceptible and therefore benefit from therapy; on the other hand, surrogate parameters (alternative measures) are defined that can be used to demonstrate efficacy.<\/p>\n<p>In all these efforts, the European Liver Registry, which is primarily based in Germany, plays a key role. Since 2015, participants have been systematically recruited and blood samples collected. These samples can be used to identify and validate the aforementioned surrogate parameters.<\/p>\n<p>For this reason, we have launched the very important longitudinal follow-up study, with which we aim to reach all participants from recent years to evaluate how the disease has progressed in the meantime. For this purpose, all participants with the most severely affected genotype Pi*ZZ will be contacted by telephone to complete a questionnaire with us.<\/p>\n<p>Of course, we need your help for this, because only in this way can we find out what has become of all the subjects who have been examined by us in the last seven years.<\/p>\n<p>Therefore, we would like to sincerely ask for your help: The approval of the first medications for alpha-1 antitrypsin deficiency-associated liver disease is within reach. Now we are all called upon to make this dream a reality \u2013 BE THERE!<\/p>\n<\/div>\n\n\n\n\n\t\t\t<\/div> \n\t\t<\/div>\n\t<\/div> \n<\/div><\/div>\n\t\t<div id=\"fws_6aa855ad6c582\"  data-column-margin=\"default\" data-midnight=\"light\" data-top-percent=\"5%\"  class=\"wpb_row vc_row-fluid vc_row\"  style=\"padding-top: calc(100vw * 0.05); padding-bottom: 0px; \"><div class=\"row-bg-wrap\" data-bg-animation=\"none\" data-bg-animation-delay=\"\" data-bg-overlay=\"false\"><div class=\"inner-wrap row-bg-layer\" ><div class=\"row-bg viewport-desktop\"  style=\"\"><\/div><\/div><\/div><div class=\"row_col_wrap_12 col span_12 light left\">\n\t<div style=\"\" class=\"vc_col-sm-12 wpb_column column_container vc_column_container col padding-3-percent inherit_tablet inherit_phone\" data-using-bg=\"true\" data-padding-pos=\"all\" data-has-bg-color=\"true\" data-bg-color=\"#004267\" data-bg-opacity=\"1\" data-animation=\"\" data-delay=\"0\" >\n\t\t<div class=\"vc_column-inner\" ><div class=\"column-bg-overlay-wrap column-bg-layer\" data-bg-animation=\"none\"><div class=\"column-bg-overlay\" style=\"opacity: 1; background-color: #004267;\"><\/div><\/div>\n\t\t\t<div class=\"wpb_wrapper\">\n\t\t\t\t\n<div class=\"wpb_text_column wpb_content_element\" >\n\t<h2>Your data is important to us!<\/h2>\n<p>We are happy to support the study centers, but we never share your data. Therefore, you may receive a letter from us, but the contents may originate from a study center. This content has been forwarded to us for delivery to you, and your data privacy remains guaranteed.<\/p>\n<\/div>\n\n\n\n\n\t\t\t<\/div> \n\t\t<\/div>\n\t<\/div> \n<\/div><\/div>","protected":false},"excerpt":{"rendered":"AutorIn Malin Fromme und Pavel Strnad H\u00e4ufig werden wir gefragt, warum es so wenige zugelassene Medikamente f\u00fcr Patienten mit Alpha-1-Antitrypsin-Mangel gibt und warum zum Beispiel die Augmentationstherapie (Ersatztherapie) nicht in...","protected":false},"author":1,"featured_media":3376,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1,39],"tags":[],"class_list":["post-5051","post","type-post","status-publish","format-standard","has-post-thumbnail","category-allgemein","category-alpha-1-leber"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Der lange Weg zu Alpha-1-Medikamenten - 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