CINXE.COM

Alnylam® RNAi Research for Patients

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class="field field--name-field-language-link field--type-entity-reference-revisions field--label-hidden field--items"> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--107 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Global</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/">English</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--108 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Austria</div> </span><span class="link-text"> <div class="field 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class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Japan</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="https://www.alnylam.jp/">日本語</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--110 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Belgium</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-belgium-dutch"> Nederlands</a></div> <div class="field--item"><a href="/alnylam-belgium-french">Français</a></div> </div> </span> 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field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="https://www.alnylam.com.br/">Português</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--111 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Netherlands </div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-netherlands">Nederlands</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--114 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Canada </div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="https://www.alnylam.ca/fr">Français </a></div> <div class="field--item"><a href="https://www.alnylam.ca/">English</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--113 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Portugal</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-portugal">Português</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--1513 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Czech Republic</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-czech-republic">Čeština</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--115 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Spain </div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-espana">Español</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--116 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Denmark </div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-denmark">Dansk</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--117 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Sweden</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-sverige">Svenska</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--118 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">France</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="https://www.alnylam.fr/">Français</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--119 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Switzerland</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-switzerland-french">Français</a></div> <div class="field--item"><a href="/alnylam-switzerland-german">Deutsch</a></div> <div class="field--item"><a href="/alnylam-switzerland-italian">Italiano</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--120 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Germany </div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="https://www.alnylam.de/">Deutsch</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--121 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Taiwan</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-taiwan">繁體中文</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--1514 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Ireland </div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-ireland">English</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--123 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">United Kingdom</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="/alnylam-uk">English</a></div> </div> </span> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--language-links paragraph-id--122 paragraph--view-mode--default"> <div class="language-switcher-main"> <span class="label-text"> <div class="field field--name-field-label field--type-string field--label-hidden field--item">Italy</div> </span><span class="link-text"> <div class="field field--name-field-link1 field--type-link field--label-hidden field--items"> <div class="field--item"><a href="https://www.alnylam.it/">Italiano</a></div> </div> </span> </div> </div> </div> </div> </section> </div> </div> </div> </div> </header> <div id="maindiv" class="container-fluid"> <main id="midcontent-area" class="column main-content"> <div id="content"> <div class="region region-content"> <div data-drupal-messages-fallback class="hidden"></div> <article data-history-node-id="8" role="article" about="/patients" typeof="schema:WebPage" class="page full clearfix"> <span property="schema:name" content="Patients" class="hidden"></span> <div class="content"> <div class="field field--name-field-add-section field--type-entity-reference-revisions field--label-hidden field--items"> <div class="field--item"> <div class="paragraph paragraph--type--sim paragraph-id--431 paragraph--view-mode--default"> <div id="simple-banner-wrapper"> <div class="simple-banner" style=""> <div class="field field--name-field-upload-image field--type-image field--label-hidden field--item"> <img src="/sites/default/files/2023-12/patients.jpg" width="1280" height="384" alt="Patients" loading="lazy" typeof="foaf:Image" class="img-responsive" /> </div> </div> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--banner-overlay-html-content paragraph-id--432 paragraph--view-mode--default"> <div class="banner-overlay-html-content-description--component " style="width:1130px; margin:auto;"> <div class="field field--name-field-body field--type-text-with-summary field--label-hidden field--item"><p class="mobile-eyebrow"><span class="our-secondary-dark">Patients</span></p> <h1 class="secondary-dark text-align-center"><strong>PATIENTS</strong></h1> <h2 class="text-align-center">Our Priority Is to Put You at the Center</h2> <p class="font-20">At Alnylam, we want to understand your journey, your experiences, and your unique needs. You inspire us to learn as much as we can, to persevere and continue to advance the development of potential therapies to treat rare and serious diseases. We take pride in working together to achieve mutual goals such as increasing awareness, enabling diagnosis, and providing education and services to support patient communities. We have a dedicated team to ensure your perspectives are represented at Alnylam. If you are an advocate interested in helping us increase awareness of rare diseases, please send an email to<a href="mailto:PatientAdvocacy@alnylam.com"> PatientAdvocacy@alnylam.com</a></p> </div> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--body paragraph-id--433 paragraph--view-mode--default"> <div class="description--component" style="width:970px; margin:auto; "> <div class="field field--name-field-body field--type-text-with-summary field--label-hidden field--item"><h2 class="text-align-center">Understanding RNAi</h2> <p class="text-align-center">RNA interference (RNAi) is the core discovery that forms the therapies Alnylam is developing. It is recognized as a major scientific breakthrough—but how does it work, exactly?</p> </div> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--video-popup paragraph-id--434 paragraph--view-mode--default"> <div class="video-popup-content" style="width:850px; margin:auto;"> <a data-colorbox-inline=".video-box-434" style="width: 571px; height:326px;"> <div class="three-column-image-box"> <div class="field field--name-field-upload-image field--type-image field--label-hidden field--item"> <img src="/sites/default/files/2021-11/For-patients_Understanding-RNAi.jpg" width="850" height="480" alt="For-patients_Understanding-RNAi" loading="lazy" typeof="foaf:Image" class="img-responsive" /> </div> <div class="three-column-video-play-button"></div> </div> <div class="video-box-434" style="display: none;"> <div class="field field--name-field-embed-video field--type-video-embed-field field--label-hidden field--item"><div class="video-embed-field-provider-youtube video-embed-field-responsive-video form-group"><iframe width="854" height="480" frameborder="0" allowfullscreen="allowfullscreen" src="https://www.youtube.com/embed/ttr_8eOjWek?autoplay=0&amp;start=0&amp;rel=0"></iframe> </div> </div> </div> </a> <div class="video-popup-desc"> <div class="field field--name-field-video-desc field--type-text-long field--label-hidden field--item"><div> <p>Learn About <strong>How RNAi-Based Medicines Work</strong></p> </div> </div> </div> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--html-content-with-background paragraph-id--436 paragraph--view-mode--default"> <div class="html-content-background " style="width:px; background-color: #f8f8f8 ;"> <div class="row inner-block" "> <div class="field field--name-field-html-content-bg field--type-entity-reference-revisions field--label-hidden field--items"> <div class="field--item"> <div class="paragraph paragraph--type--body paragraph-id--435 paragraph--view-mode--default"> <div class="description--component" style="width:1132px; margin:auto; "> <div class="field field--name-field-body field--type-text-with-summary field--label-hidden field--item"><div class="advocacy-bottom-grid"> <div class="row"> <div class="col-sm-3"> <div class="grid-img"><img alt="Aln_Icons_RNAi" data-entity-type="file" data-entity-uuid="05609c1e-dd8d-4349-b368-2cddbd976284" src="/sites/default/files/inline-images/Aln_Icons_RNAi.png" width="147" height="147" loading="lazy" /></div> <div class="grid-text"> <p>Learn about RNAi and our investigational therapies</p> <a href="/our-science/the-science-of-rnai/">Read More ›</a></div> </div> <div class="col-sm-3"> <div class="grid-img"><img alt="Aln_Icons_4Stars" data-entity-type="file" data-entity-uuid="e18e556b-0a23-48a9-a42a-fbe15db48aaa" src="/sites/default/files/inline-images/Aln_Icons_4Stars.png" width="147" height="147" loading="lazy" /></div> <div class="grid-text"> <p>Alnylam is focused on several disease areas including transthyretin amyloidosis, rare diseases, cardiovascular diseases, metabolic diseases, and neurological diseases.</p> <p><a href="/alnylam-rnai-pipeline/">Start Here ›</a></p> </div> </div> <div class="col-sm-3"> <div class="grid-img"><img alt="Caregivers" data-entity-type="file" data-entity-uuid="5f014f21-5fc9-49ee-b377-f52151b475eb" src="/sites/default/files/inline-images/Caregivers.png" width="147" height="147" loading="lazy" /></div> <div class="grid-text"> <p>Alnylam is dedicated to providing support for affected individuals, families, and caregivers</p> <p><a href="/patients/patient-services">About Alnylam Patient Services ›</a></p> </div> </div> <div class="col-sm-3"> <div class="grid-img"><img alt="Aln_Icons_Interested2" data-entity-type="file" data-entity-uuid="de059500-c51c-4cf3-a306-e358ce26c9d9" src="/sites/default/files/inline-images/Aln_Icons_Interested2.png" width="147" height="147" loading="lazy" /></div> <div class="grid-text"> <p>Interested in our clinical trials?</p> <p><a href="/our-pipeline/clinical-trials/">Learn More ›</a></p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--_-column-video-section paragraph-id--440 paragraph--view-mode--default"> <div class="three-column-video-desc-box" style="width:1132px; margin:auto; "> <div class="field field--name-field-body field--type-text-with-summary field--label-hidden field--item"><h2 class="text-align-center">Our Focus On You</h2> </div> </div> <div class="three-column-video-content-box" style="width:1132px; margin:auto; "> <div class="field field--name-field-3-column-video-card field--type-entity-reference-revisions field--label-hidden field--items"> <div class="field--item"> <div class="container col-sm-4"> <div class="paragraph paragraph--type--heading-video-description paragraph-id--437 paragraph--view-mode--default"> <div class="three-column-video-content"> <a data-colorbox-inline=".three-column-video-box-437" style="width: 650px; height:400px;"> <div class="three-column-image-box"> <div class="field field--name-field-upload-image field--type-image field--label-hidden field--item"> <img src="/sites/default/files/styles/three_column_image/public/2021-11/Journey_to_Diagnosis_2_0.jpg?itok=f6I8HTac" width="358" height="300" alt="Journey_to_Diagnosis_2_0" loading="lazy" typeof="foaf:Image" class="img-responsive" /> </div> <div class="three-column-video-play-button"> <img class="normal-video" src="/themes/custom/alnylam/images/play-button-56-px.svg"> <img class="active-video" src="/themes/custom/alnylam/images/play-button-active-56-px.svg"> </div> </div> <div class="three-column-video-box-437" style="display: none;"> <div class="field field--name-field-embed-video field--type-video-embed-field field--label-hidden field--item"><div class="video-embed-field-provider-youtube video-embed-field-responsive-video form-group"><iframe width="854" height="480" frameborder="0" allowfullscreen="allowfullscreen" src="https://www.youtube.com/embed/c0P99mmiI50?autoplay=0&amp;start=0&amp;rel=0"></iframe> </div> </div> </div> </a> </div> <div class="three-column-title-and-width-box"> <div class="three-column-video-heading"> <div class="field field--name-field-heading-1 field--type-string field--label-hidden field--item">A Family Living with</div> </div> <div class="three-column-video-heading2"> <div class="field field--name-field-heading-2 field--type-string field--label-hidden field--item"> hATTR Amyloidosis</div> </div> </div> <div class="three-column-video-short-description"> <div class="field field--name-field-short-description field--type-string field--label-hidden field--item">Get more information about hATTR Amyloidosis.</div> </div> <div class="three-column-video-link"> <div class="field field--name-field-link field--type-link field--label-hidden field--item"><a href="/patients/amyloidosis">Start Here ›</a></div> </div> </div> </div> </div> <div class="field--item"> <div class="container col-sm-4"> <div class="paragraph paragraph--type--heading-video-description paragraph-id--438 paragraph--view-mode--default"> <div class="three-column-video-content"> <a data-colorbox-inline=".three-column-video-box-438" style="width: 650px; height:400px;"> <div class="three-column-image-box"> <div class="field field--name-field-upload-image field--type-image field--label-hidden field--item"> <img src="/sites/default/files/styles/three_column_image/public/2021-11/ania_0.jpg?itok=bwGZqZbo" width="358" height="300" alt="ania_0.jpg" loading="lazy" typeof="foaf:Image" class="img-responsive" /> </div> <div class="three-column-video-play-button"> <img class="normal-video" src="/themes/custom/alnylam/images/play-button-56-px.svg"> <img class="active-video" src="/themes/custom/alnylam/images/play-button-active-56-px.svg"> </div> </div> <div class="three-column-video-box-438" style="display: none;"> <div class="field field--name-field-embed-video field--type-video-embed-field field--label-hidden field--item"><div class="video-embed-field-provider-youtube video-embed-field-responsive-video form-group"><iframe width="854" height="480" frameborder="0" allowfullscreen="allowfullscreen" src="https://www.youtube.com/embed/3ib8b-HW4zk?autoplay=0&amp;start=0&amp;rel=0"></iframe> </div> </div> </div> </a> </div> <div class="three-column-title-and-width-box"> <div class="three-column-video-heading"> <div class="field field--name-field-heading-1 field--type-string field--label-hidden field--item">Ania: Living with</div> </div> <div class="three-column-video-heading2"> <div class="field field--name-field-heading-2 field--type-string field--label-hidden field--item">Porphyria</div> </div> </div> <div class="three-column-video-short-description"> <div class="field field--name-field-short-description field--type-string field--label-hidden field--item">Get more information about porphyria.</div> </div> <div class="three-column-video-link"> <div class="field field--name-field-link field--type-link field--label-hidden field--item"><a href="/patients/porphyria">Start Here ›</a></div> </div> </div> </div> </div> <div class="field--item"> <div class="container col-sm-4"> <div class="paragraph paragraph--type--heading-video-description paragraph-id--439 paragraph--view-mode--default"> <div class="three-column-video-content"> <a data-colorbox-inline=".three-column-video-box-439" style="width: 650px; height:400px;"> <div class="three-column-image-box"> <div class="field field--name-field-upload-image field--type-image field--label-hidden field--item"> <img src="/sites/default/files/styles/three_column_image/public/2021-11/Alnylam_PH1_video358x301_L2_0.jpg?itok=LGok04ol" width="358" height="300" alt="Alnylam_PH1_video358x301_L2_0.jpg" loading="lazy" typeof="foaf:Image" class="img-responsive" /> </div> <div class="three-column-video-play-button"> <img class="normal-video" src="/themes/custom/alnylam/images/play-button-56-px.svg"> <img class="active-video" src="/themes/custom/alnylam/images/play-button-active-56-px.svg"> </div> </div> <div class="three-column-video-box-439" style="display: none;"> <div class="field field--name-field-embed-video field--type-video-embed-field field--label-hidden field--item"><div class="video-embed-field-provider-youtube video-embed-field-responsive-video form-group"><iframe width="854" height="480" frameborder="0" allowfullscreen="allowfullscreen" src="https://www.youtube.com/embed/8ZKPm2--0CY?autoplay=0&amp;start=0&amp;rel=0"></iframe> </div> </div> </div> </a> </div> <div class="three-column-title-and-width-box"> <div class="three-column-video-heading"> <div class="field field--name-field-heading-1 field--type-string field--label-hidden field--item">A Family Living with </div> </div> <div class="three-column-video-heading2"> <div class="field field--name-field-heading-2 field--type-string field--label-hidden field--item">Primary Hyperoxaluria Type 1 (PH1)</div> </div> </div> <div class="three-column-video-short-description"> <div class="field field--name-field-short-description field--type-string field--label-hidden field--item">Get more information about primary hyperoxaluria.</div> </div> <div class="three-column-video-link"> <div class="field field--name-field-link field--type-link field--label-hidden field--item"><a href="/patients/primary-hyperoxaluria">Start Here ›</a></div> </div> </div> </div> </div> </div> </div> </div> </div></div> <div class="field--item"> <div class="paragraph paragraph--type--dark-bg-banner paragraph-id--443 paragraph--view-mode--default" style="background-color:#00254b;" > <div id="dark-bg-banner" style="width:1170px; margin:auto;"> <div class="row"> <div class="field field--name-field-description-text-area field--type-text-long field--label-hidden field--item"><div class="row"> <div class="col-sm-9"> <h2>Access To Investigational Medicines</h2> <p>Alnylam is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness. We understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Alnylam will consider providing a requesting physician with pre-approval access to a specific Alnylam investigational medicine, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.</p> </div> <div class="col-sm-3" style="text-align: right;"><a class="button-more" href="/sites/default/files/pdfs/Alnylam-Compassionate-Use-Policy.pdf" target="_blank">Learn More ›</a></div> </div> </div> </div> </div> </div> </div> <div class="field--item"> <div class="paragraph paragraph--type--_-column-heading-image-descripti paragraph-id--448 paragraph--view-mode--default"> <div style="background:; overflow:hidden; padding: 34px 0px 64px 0px" class="patient-philosophy"> <div style="width:1100px; margin:auto;"> <div class="field field--name-field-2-column-pop-content field--type-entity-reference-revisions field--label-hidden field--items"> <div class="field--item"> <div class="paragraph 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