{"id":6534,"date":"2023-04-18T20:05:41","date_gmt":"2023-04-18T20:05:41","guid":{"rendered":"https:\/\/restruct-lgmd-2021.pantheonsite.io\/?page_id=6534"},"modified":"2026-03-29T20:09:57","modified_gmt":"2026-03-29T20:09:57","slug":"clinical-trials-resources","status":"publish","type":"page","link":"https:\/\/www.lgmd-info.org\/ru\/knowledge-base\/navigating-lgmd\/for-patients\/clinical-trials-resources\/","title":{"rendered":"Clinical Trials Resources"},"content":{"rendered":"<p><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-1 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-0 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:20px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-1\"><div id=\"lipsum\">\n<div class=\"group w-full text-token-text-primary border-b border-black\/10 dark:border-gray-900\/50 bg-gray-50 dark:bg-&#091;#444654&#093;\">\n<div class=\"flex p-4 gap-4 text-base md:gap-6 md:max-w-2xl lg:max-w-&#091;38rem&#093; xl:max-w-3xl md:py-6 lg:px-0 m-auto\">\n<div class=\"relative flex w-&#091;calc(100%-50px)&#093; flex-col gap-1 md:gap-3 lg:w-&#091;calc(100%-115px)&#093;\">\n<div class=\"flex flex-grow flex-col gap-3\">\n<div class=\"min-h-&#091;20px&#093; flex flex-col items-start gap-3 overflow-x-auto whitespace-pre-wrap break-words\">\n<div class=\"markdown prose w-full break-words dark:prose-invert dark\">\n<p>Navigating the world of clinical trials can be complex, especially for those diagnosed with limb-girdle muscular dystrophy (LGMD). The &#8220;Clinical Trials Resources&#8221; page is designed to simplify this journey, offering a wealth of information and resources tailored specifically for LGMD patients. Whether you&#8217;re considering participating in a trial or simply seeking to understand the latest research developments, this guide provides clarity and direction, ensuring you&#8217;re well-informed every step of the way.<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-2 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-1 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-overflow:hidden;--awb-bg-color:var(--awb-color4);--awb-bg-color-hover:var(--awb-color4);--awb-bg-size:cover;--awb-box-shadow:0px 0px 4px 0px var(--awb-color2);;--awb-border-radius:5px 5px 5px 5px;--awb-width-large:100%;--awb-margin-top-large:20px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:20px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-title title fusion-title-1 fusion-sep-none fusion-title-text fusion-title-size-three\" style=\"--awb-text-color:var(--awb-color1);--awb-margin-right:10px;--awb-margin-bottom:10px;--awb-margin-left:15px;--awb-font-size:var(--awb-custom_typography_3-font-size);\"><h3 class=\"fusion-title-heading title-heading-left fusion-responsive-typography-calculated\" style=\"font-family:var(--awb-custom_typography_3-font-family);font-weight:var(--awb-custom_typography_3-font-weight);font-style:var(--awb-custom_typography_3-font-style);margin:0;font-size:1em;letter-spacing:var(--awb-custom_typography_3-letter-spacing);text-transform:var(--awb-custom_typography_3-text-transform);--fontSize:26;--minFontSize:26;line-height:var(--awb-custom_typography_3-line-height);\">LGMD Clinical Trials &amp; Natural History Studies<\/h3><\/div><div class=\"fusion-separator fusion-full-width-sep\" style=\"align-self: center;margin-left: auto;margin-right: auto;width:100%;\"><div class=\"fusion-separator-border sep-shadow\" style=\"--awb-height:20px;--awb-amount:20px;--awb-sep-color:var(--awb-color1);background:radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-webkit-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-moz-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-o-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);\"><\/div><\/div><div class=\"fusion-builder-row fusion-builder-row-inner fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"width:104% !important;max-width:104% !important;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column_inner fusion-builder-nested-column-0 fusion_builder_column_inner_1_1 1_1 fusion-flex-column\" style=\"--awb-padding-top:15px;--awb-padding-right:15px;--awb-padding-bottom:15px;--awb-padding-left:15px;--awb-bg-color:#f2f2f2;--awb-bg-color-hover:#f2f2f2;--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-2 fusion-text-no-margin\" style=\"--awb-font-size:var(--awb-custom_typography_6-font-size);--awb-line-height:var(--awb-custom_typography_6-line-height);--awb-letter-spacing:var(--awb-custom_typography_6-letter-spacing);--awb-text-transform:var(--awb-custom_typography_6-text-transform);--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-text-font-family:var(--awb-custom_typography_6-font-family);--awb-text-font-weight:var(--awb-custom_typography_6-font-weight);--awb-text-font-style:var(--awb-custom_typography_6-font-style);\"><p>Clinical Trials and Natural History Studies are vital for advancing research to help find a treatment and cure for LGMD.<\/p>\n<\/div><ul style=\"--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-iconcolor:var(--awb-color4);--awb-line-height:27.2px;--awb-icon-width:27.2px;--awb-icon-height:27.2px;--awb-icon-margin:11.2px;--awb-content-margin:38.4px;--awb-circlecolor:rgba(255,255,255,0);--awb-circle-yes-font-size:14.08px;\" class=\"fusion-checklist fusion-checklist-1 fusion-checklist-default type-icons\"><li class=\"fusion-li-item\" style=\"\"><span class=\"icon-wrapper circle-yes\"><i class=\"fusion-li-icon fa-check fas\" aria-hidden=\"true\"><\/i><\/span><div class=\"fusion-li-item-content\">\n<p><strong><a href=\"https:\/\/clinicaltrials.gov\/\">ClinicalTrials.gov<\/a>\u00a0-\u00a0<\/strong>This is a searchable registry and results database of federally and privately supported clinical trials conducted in the United States and around the world.<\/p>\n<\/div><\/li><li class=\"fusion-li-item\" style=\"\"><span class=\"icon-wrapper circle-yes\"><i class=\"fusion-li-icon fa-check fas\" aria-hidden=\"true\"><\/i><\/span><div class=\"fusion-li-item-content\">\n<p>Biotech and Pharmaceutical Companies<\/p>\n<ul>\n<li><a href=\"https:\/\/www.askbio.com\/genetic-disease-focus\/\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>AskBio&nbsp;<\/strong><\/a><\/li>\n<li><a href=\"https:\/\/atamyo.com\/\" target=\"_blank\" rel=\"noopener\"><strong>Atamyo Therapeutics&nbsp;<\/strong><\/a><\/li>\n<li><a href=\"https:\/\/bridgebio.com\/\" target=\"_blank\" rel=\"noopener\"><strong>BridgeBio<\/strong><\/a><\/li>\n<li><strong><a href=\"https:\/\/edgewisetx.com\/\" target=\"_blank\" rel=\"noopener\">Edgewise Therapeutics<\/a><\/strong><\/li>\n<li><strong><a href=\"https:\/\/www.genethon.fr\/en\/products\/limb-girdle-muscular-dystrophy-with-fkrp-deficiency\/\" target=\"_blank\" rel=\"noopener noreferrer\">Genethon<\/a><\/strong><\/li>\n<li><a href=\"https:\/\/kineabio.com\/\" target=\"_blank\" rel=\"noopener\"><strong>Kinea Bio<\/strong><\/a><\/li>\n<li><a href=\"https:\/\/www.sarepta.com\/disease-areas\/limb-girdle-muscular-dystrophy\" target=\"_blank\" rel=\"noopener noreferrer\"><strong>\u0421\u0430\u0440\u0435\u043f\u0442\u0430 \u0422\u0435\u0440\u0430\u043f\u044c\u044e\u0442\u0438\u043a\u0441<\/strong><\/a><\/li>\n<\/ul>\n<\/div><\/li><\/ul><\/div><\/div><\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-3 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-2 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-overflow:hidden;--awb-bg-color:var(--awb-color4);--awb-bg-color-hover:var(--awb-color4);--awb-bg-size:cover;--awb-box-shadow:0px 0px 4px 0px var(--awb-color2);;--awb-border-radius:5px 5px 5px 5px;--awb-width-large:100%;--awb-margin-top-large:20px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:20px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-title title fusion-title-2 fusion-sep-none fusion-title-text fusion-title-size-three\" style=\"--awb-text-color:var(--awb-color1);--awb-margin-right:10px;--awb-margin-bottom:10px;--awb-margin-left:15px;--awb-font-size:var(--awb-custom_typography_3-font-size);\"><h3 class=\"fusion-title-heading title-heading-left fusion-responsive-typography-calculated\" style=\"font-family:var(--awb-custom_typography_3-font-family);font-weight:var(--awb-custom_typography_3-font-weight);font-style:var(--awb-custom_typography_3-font-style);margin:0;font-size:1em;letter-spacing:var(--awb-custom_typography_3-letter-spacing);text-transform:var(--awb-custom_typography_3-text-transform);--fontSize:26;--minFontSize:26;line-height:var(--awb-custom_typography_3-line-height);\">Rare Research Map<\/h3><\/div><div class=\"fusion-separator fusion-full-width-sep\" style=\"align-self: center;margin-left: auto;margin-right: auto;width:100%;\"><div class=\"fusion-separator-border sep-shadow\" style=\"--awb-height:20px;--awb-amount:20px;--awb-sep-color:var(--awb-color1);background:radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-webkit-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-moz-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-o-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);\"><\/div><\/div><div class=\"fusion-builder-row fusion-builder-row-inner fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"width:104% !important;max-width:104% !important;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column_inner fusion-builder-nested-column-1 fusion_builder_column_inner_1_1 1_1 fusion-flex-column\" style=\"--awb-padding-top:15px;--awb-padding-right:15px;--awb-padding-bottom:15px;--awb-padding-left:15px;--awb-bg-color:#f2f2f2;--awb-bg-color-hover:#f2f2f2;--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-3 fusion-text-no-margin\" style=\"--awb-font-size:var(--awb-custom_typography_6-font-size);--awb-line-height:var(--awb-custom_typography_6-line-height);--awb-letter-spacing:var(--awb-custom_typography_6-letter-spacing);--awb-text-transform:var(--awb-custom_typography_6-text-transform);--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-text-font-family:var(--awb-custom_typography_6-font-family);--awb-text-font-weight:var(--awb-custom_typography_6-font-weight);--awb-text-font-style:var(--awb-custom_typography_6-font-style);\"><p>Following the 2022 Rare Drug Development Symposium, Global Genes released the Rare Disease Roadmap. This toolkit was designed to help advocates, individuals, families, and organizations better understand some of the potential routes to treatment and some of the key concepts that are part of these processes to work with researchers and industry partners throughout the drug development process.<\/p>\n<\/div><ul style=\"--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-iconcolor:var(--awb-color4);--awb-line-height:27.2px;--awb-icon-width:27.2px;--awb-icon-height:27.2px;--awb-icon-margin:11.2px;--awb-content-margin:38.4px;--awb-circlecolor:rgba(255,255,255,0);--awb-circle-yes-font-size:14.08px;\" class=\"fusion-checklist fusion-checklist-2 fusion-checklist-default type-icons\"><li class=\"fusion-li-item\" style=\"\"><span class=\"icon-wrapper circle-yes\"><i class=\"fusion-li-icon fa-check fas\" aria-hidden=\"true\"><\/i><\/span><div class=\"fusion-li-item-content\">\n<p><a href=\"https:\/\/www.lgmd-info.org\/wp-content\/uploads\/2023\/01\/Rare-Research-Roadmap.pdf\" target=\"_blank\" rel=\"noopener\"><strong>Rare Research Roadmap<\/strong><\/a><\/p>\n<\/div><\/li><\/ul><\/div><\/div><\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-4 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-3 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-overflow:hidden;--awb-bg-color:var(--awb-color4);--awb-bg-color-hover:var(--awb-color4);--awb-bg-size:cover;--awb-box-shadow:0px 0px 4px 0px var(--awb-color2);;--awb-border-radius:5px 5px 5px 5px;--awb-width-large:100%;--awb-margin-top-large:20px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:20px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-title title fusion-title-3 fusion-sep-none fusion-title-text fusion-title-size-three\" style=\"--awb-text-color:var(--awb-color1);--awb-margin-right:10px;--awb-margin-bottom:10px;--awb-margin-left:15px;--awb-font-size:var(--awb-custom_typography_3-font-size);\"><h3 class=\"fusion-title-heading title-heading-left fusion-responsive-typography-calculated\" style=\"font-family:var(--awb-custom_typography_3-font-family);font-weight:var(--awb-custom_typography_3-font-weight);font-style:var(--awb-custom_typography_3-font-style);margin:0;font-size:1em;letter-spacing:var(--awb-custom_typography_3-letter-spacing);text-transform:var(--awb-custom_typography_3-text-transform);--fontSize:26;--minFontSize:26;line-height:var(--awb-custom_typography_3-line-height);\">Checklist for Assessing the Legitimacy of Any Proposed LGMD Treatment<\/h3><\/div><div class=\"fusion-separator fusion-full-width-sep\" style=\"align-self: center;margin-left: auto;margin-right: auto;width:100%;\"><div class=\"fusion-separator-border sep-shadow\" style=\"--awb-height:20px;--awb-amount:20px;--awb-sep-color:var(--awb-color1);background:radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-webkit-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-moz-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-o-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);\"><\/div><\/div><div class=\"fusion-builder-row fusion-builder-row-inner fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"width:104% !important;max-width:104% !important;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column_inner fusion-builder-nested-column-2 fusion_builder_column_inner_1_1 1_1 fusion-flex-column\" style=\"--awb-padding-top:15px;--awb-padding-right:15px;--awb-padding-bottom:15px;--awb-padding-left:15px;--awb-bg-color:#f2f2f2;--awb-bg-color-hover:#f2f2f2;--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-4 fusion-text-no-margin\" style=\"--awb-font-size:var(--awb-custom_typography_6-font-size);--awb-line-height:var(--awb-custom_typography_6-line-height);--awb-letter-spacing:var(--awb-custom_typography_6-letter-spacing);--awb-text-transform:var(--awb-custom_typography_6-text-transform);--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-text-font-family:var(--awb-custom_typography_6-font-family);--awb-text-font-weight:var(--awb-custom_typography_6-font-weight);--awb-text-font-style:var(--awb-custom_typography_6-font-style);\"><p>Patients can empower themselves by investigating whether or not a treatment has been published and proven to be safe and effective. \u00a0\u00a0For the health &amp; safety of the LGMD patient, it is extremely important to determine whether a treatment is legitimate!<\/p>\n<\/div><ul style=\"--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-iconcolor:var(--awb-color4);--awb-line-height:27.2px;--awb-icon-width:27.2px;--awb-icon-height:27.2px;--awb-icon-margin:11.2px;--awb-content-margin:38.4px;--awb-circlecolor:rgba(255,255,255,0);--awb-circle-yes-font-size:14.08px;\" class=\"fusion-checklist fusion-checklist-3 fusion-checklist-default type-icons\"><li class=\"fusion-li-item\" style=\"\"><span class=\"icon-wrapper circle-yes\"><i class=\"fusion-li-icon fa-check fas\" aria-hidden=\"true\"><\/i><\/span><div class=\"fusion-li-item-content\">\n<p><a href=\"https:\/\/www.lgmd-info.org\/ru\/in-search-of-treatment-or-a-cure-for-lgmd-3\/\" target=\"_blank\" rel=\"noopener\"><strong>5 Question \u201cChecklist\u201d<\/strong>\u00a0<\/a>(PDF Document)<\/p>\n<\/div><\/li><\/ul><\/div><\/div><\/div><\/div><\/div><\/div><\/div><div class=\"fusion-fullwidth fullwidth-box fusion-builder-row-5 fusion-flex-container has-pattern-background has-mask-background nonhundred-percent-fullwidth non-hundred-percent-height-scrolling\" style=\"--awb-border-radius-top-left:0px;--awb-border-radius-top-right:0px;--awb-border-radius-bottom-right:0px;--awb-border-radius-bottom-left:0px;--awb-flex-wrap:wrap;\" ><div class=\"fusion-builder-row fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"max-width:1456px;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column fusion-builder-column-4 fusion_builder_column_1_1 1_1 fusion-flex-column\" style=\"--awb-overflow:hidden;--awb-bg-color:var(--awb-color4);--awb-bg-color-hover:var(--awb-color4);--awb-bg-size:cover;--awb-box-shadow:0px 0px 4px 0px var(--awb-color2);;--awb-border-radius:5px 5px 5px 5px;--awb-width-large:100%;--awb-margin-top-large:20px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:20px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-title title fusion-title-4 fusion-sep-none fusion-title-text fusion-title-size-three\" style=\"--awb-text-color:var(--awb-color1);--awb-margin-right:10px;--awb-margin-bottom:10px;--awb-margin-left:15px;--awb-font-size:var(--awb-custom_typography_3-font-size);\"><h3 class=\"fusion-title-heading title-heading-left fusion-responsive-typography-calculated\" style=\"font-family:var(--awb-custom_typography_3-font-family);font-weight:var(--awb-custom_typography_3-font-weight);font-style:var(--awb-custom_typography_3-font-style);margin:0;font-size:1em;letter-spacing:var(--awb-custom_typography_3-letter-spacing);text-transform:var(--awb-custom_typography_3-text-transform);--fontSize:26;--minFontSize:26;line-height:var(--awb-custom_typography_3-line-height);\">Glossary of Drug Development Terms<\/h3><\/div><div class=\"fusion-separator fusion-full-width-sep\" style=\"align-self: center;margin-left: auto;margin-right: auto;width:100%;\"><div class=\"fusion-separator-border sep-shadow\" style=\"--awb-height:20px;--awb-amount:20px;--awb-sep-color:var(--awb-color1);background:radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-webkit-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-moz-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);background:-o-radial-gradient(ellipse at 50% -50% , var(--awb-color1) 0px, rgba(255, 255, 255, 0) 80%) repeat scroll 0 0 rgba(0, 0, 0, 0);\"><\/div><\/div><div class=\"fusion-builder-row fusion-builder-row-inner fusion-row fusion-flex-align-items-flex-start fusion-flex-content-wrap\" style=\"width:104% !important;max-width:104% !important;margin-left: calc(-4% \/ 2 );margin-right: calc(-4% \/ 2 );\"><div class=\"fusion-layout-column fusion_builder_column_inner fusion-builder-nested-column-3 fusion_builder_column_inner_1_1 1_1 fusion-flex-column\" style=\"--awb-padding-top:15px;--awb-padding-right:15px;--awb-padding-bottom:15px;--awb-padding-left:15px;--awb-bg-color:#f2f2f2;--awb-bg-color-hover:#f2f2f2;--awb-bg-size:cover;--awb-width-large:100%;--awb-margin-top-large:0px;--awb-spacing-right-large:1.92%;--awb-margin-bottom-large:0px;--awb-spacing-left-large:1.92%;--awb-width-medium:100%;--awb-order-medium:0;--awb-spacing-right-medium:1.92%;--awb-spacing-left-medium:1.92%;--awb-width-small:100%;--awb-order-small:0;--awb-spacing-right-small:1.92%;--awb-spacing-left-small:1.92%;\"><div class=\"fusion-column-wrapper fusion-column-has-shadow fusion-flex-justify-content-flex-start fusion-content-layout-column\"><div class=\"fusion-text fusion-text-5 fusion-text-no-margin\" style=\"--awb-font-size:var(--awb-custom_typography_6-font-size);--awb-line-height:var(--awb-custom_typography_6-line-height);--awb-letter-spacing:var(--awb-custom_typography_6-letter-spacing);--awb-text-transform:var(--awb-custom_typography_6-text-transform);--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-text-font-family:var(--awb-custom_typography_6-font-family);--awb-text-font-weight:var(--awb-custom_typography_6-font-weight);--awb-text-font-style:var(--awb-custom_typography_6-font-style);\"><p>In discussing or reading about the drug development processes, you will likely encounter references to dozens of technical terms, often with the authors&#8217; assumption that the reader will know their meaning and what their various abbreviations and acronyms stand for.  To assist you with the &#8220;alphabet soup,&#8221; our friends at the Jain Foundation have prepared this glossary of drug development terms for us to share.<\/p>\n<\/div><ul style=\"--awb-margin-top:10px;--awb-margin-bottom:10px;--awb-iconcolor:var(--awb-color4);--awb-line-height:27.2px;--awb-icon-width:27.2px;--awb-icon-height:27.2px;--awb-icon-margin:11.2px;--awb-content-margin:38.4px;--awb-circlecolor:rgba(255,255,255,0);--awb-circle-yes-font-size:14.08px;\" class=\"fusion-checklist fusion-checklist-4 fusion-checklist-default type-icons\"><li class=\"fusion-li-item\" style=\"\"><span class=\"icon-wrapper circle-yes\"><i class=\"fusion-li-icon fa-check fas\" aria-hidden=\"true\"><\/i><\/span><div class=\"fusion-li-item-content\">\n<p><a href=\"https:\/\/www.lgmd-info.org\/wp-content\/uploads\/2025\/03\/Glossary-of-Drug-Development-Terms_.pdf\" target=\"_blank\" rel=\"noopener\"><strong>Glossary of Drug Development Terms<\/strong><\/a> (PDF Document)<\/p>\n<\/div><\/li><\/ul><\/div><\/div><\/div><\/div><\/div><\/div><\/div><\/p>","protected":false},"excerpt":{"rendered":"","protected":false},"author":1,"featured_media":0,"parent":6524,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-6534","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v23.9 - 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