{"id":4924,"date":"2025-09-02T09:11:18","date_gmt":"2025-09-02T09:11:18","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/glossary\/"},"modified":"2026-01-07T19:32:14","modified_gmt":"2026-01-07T19:32:14","slug":"glossary","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/glossary\/","title":{"rendered":"Glossary"},"content":{"rendered":"<div class=\"cm-glossary\"><div class=\"glossary-container \"><input type=\"hidden\" class=\"cmtt-attribute-field\" name=\"glossary_index_style\" value=\"classic\"><div id=\"glossaryList-nav\" class=\"listNav small\" role=\"tablist\"><\/div><ul class=\"glossaryList\" role=\"tablist\" id=\"glossaryList\" itemscope itemtype=\"https:\/\/schema.org\/DefinedTermSet\"><li class=\"\" itemscope itemtype=\"https:\/\/schema.org\/DefinedTerm\" role=\"tab\"><a href=\"https:\/\/medical.biomarin.com\/en-us\/glossary\/antisense-oligonucleotides\/\" role=\"term\" class=\"glossaryLink glossary-link-title \" style=\"\" itemprop=\"url\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Antisense oligonucleotides&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Antisense oligonucleotide therapy involves the use of short, single-stranded pieces of genetic material called oligonucleotides to selectively target and modify the splicing of RNA molecules within cells. By binding to specific RNA sequences, these oligonucleotides can influence gene expression. Antisense oligonucleotide therapy holds potential for treating genetic disorders and other conditions by modulating the production of disease-associated proteins.&amp;lt;sup&amp;gt;8,9&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"><span itemprop=\"name\">Antisense oligonucleotides<\/span><\/a><\/li><li class=\"\" itemscope itemtype=\"https:\/\/schema.org\/DefinedTerm\" role=\"tab\"><a href=\"https:\/\/medical.biomarin.com\/en-us\/glossary\/biologics\/\" role=\"term\" class=\"glossaryLink glossary-link-title \" style=\"\" itemprop=\"url\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Biologics&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Biologic therapy involves the use of living organisms or their products to treat various medical conditions. These therapies often include proteins, biologics, or other complex molecules that are engineered to target specific disease processes in the body. Biologic therapies have revolutionized the treatment landscape, offering innovative approaches for conditions such as autoimmune diseases, cancer, and chronic inflammatory disorders.&amp;lt;sup&amp;gt;10,11&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"><span itemprop=\"name\">Biologics<\/span><\/a><\/li><li class=\"\" itemscope itemtype=\"https:\/\/schema.org\/DefinedTerm\" role=\"tab\"><a href=\"https:\/\/medical.biomarin.com\/en-us\/glossary\/gene-therapies\/\" role=\"term\" class=\"glossaryLink glossary-link-title \" style=\"\" itemprop=\"url\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Gene therapies&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Gene therapy aims to treat or correct genetic disorders by introducing or modifying genetic material within a person&amp;#039;s cells. This is achieved by transferring therapeutic genes into the body to replace faulty or missing genes, allowing cells to produce functional proteins and restore normal cellular function. &amp;lt;sup&amp;gt;5&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"><span itemprop=\"name\">Gene therapies<\/span><\/a><\/li><li class=\"\" itemscope itemtype=\"https:\/\/schema.org\/DefinedTerm\" role=\"tab\"><a href=\"https:\/\/medical.biomarin.com\/en-us\/glossary\/small-molecules\/\" role=\"term\" class=\"glossaryLink glossary-link-title \" style=\"\" itemprop=\"url\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Small molecules&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Small molecule therapy refers to a type of medical treatment that utilizes small, chemically synthesized compounds to interact with specific proteins or enzymes in the body. These molecules can modulate cellular pathways, signaling, or biochemical processes. Small molecule therapy is a versatile approach used to target a wide range of diseases, including infectious and metabolic disorders.&amp;lt;sup&amp;gt;6,7&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"><span itemprop=\"name\">Small molecules<\/span><\/a><\/li><\/ul><\/div><\/div>\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-4924","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Glossary | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"View the BioMarin Medical glossary\" 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