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id="main" class="home-content page-content" role="main"><a id="main-content"></a> <div class="page-header-section"> <div class="container" id="header-prewrapper"> <div id="home-hero"> <div class="frame-wrapper"> <div class="frame-placeholder"><img alt="" class="img-responsive" src="/themes/onp/img/hero/empty-frame-big.png" /></div> <div class="scene scene-1"><img alt="A treatment for the polyneuropathy of hATTR amyloidosis in adults" class="sky" src="/themes/onp/img/hero/sky-1-big.png"/> <img alt="A treatment for the polyneuropathy of hATTR amyloidosis in adults" class="ground" src="/themes/onp/img/hero/ground-1-big.png"/> <img alt="A treatment for the polyneuropathy of hATTR amyloidosis in adults" class="tree" src="/themes/onp/img/hero/unhealthy-tree-big.png"/> <div class="copy hidden-xs d-none d-md-block"> <div class="container-fluid"> <div class="row"> <div class="offset-md-5 col-md-7 offset-lg-5 col-lg-7"> <p class="head-text"><span class="font-bold">With hereditary transthyretin-mediated (hATTR) amyloidosis…</span> </p> </div> <div class="offset-md-5 col-md-7 offset-lg-5 col-lg-7"> <div class="subheader-content-wrapper"> <p class="subhead-text">Patients and their families face a future of functional decline<sup>1-3</sup> </p> </div> </div> </div> </div> </div> </div> <div class="scene scene-2"><img alt="A treatment for the polyneuropathy of hATTR amyloidosis in adults" class="sky" src="/themes/onp/img/hero/sky-2-big.png"/> <img alt="A treatment for the polyneuropathy of hATTR amyloidosis in adults" class="ground" src="/themes/onp/img/hero/ground-2-big.png"/> <img alt="A treatment for the polyneuropathy of hATTR amyloidosis in adults" class="tree" src="/themes/onp/img/hero/healthy-tree-us-big.png"/> <div class="copy hidden-xs d-none d-md-block"> <div class="container-fluid" id="header-wrapper"> <div class="row"> <div class="offset-md-5 col-md-70 offset-lg-5 col-lg-7"> <p class="head-text"><span class="font-bold">ONPATTRO<sup>®</sup> (patisiran)</span> can reverse polyneuropathy manifestations of the disease<sup>4,5</sup> </p> </div> <div class="offset-md-5 col-md-7 offset-lg-5 col-lg-7"> <div class="subheader-content-wrapper"> <p class="subhead-text font-bold">ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.</p> <p class="subhead-text font-bold">At 18 months in a placebo-controlled study, ONPATTRO demonstrated<sup>4</sup>:</p> <ul class="leaf-purple-list"> <li>Reversal in neuropathy impairment from baseline as measured by modified Neuropathy Impairment Score + 7 (mNIS+7) </li> <li>Improvement in quality of life from baseline as measured by Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) score </li> </ul> <button type="button" class="btn btn-primary link-container" id="efficacy-data"><a href="/efficacy">See efficacy data</a> </button> </div> </div> </div> </div> </div> </div> </div> <div class="copy-mobile visible-xs-block mt-15 d-block d-md-none"> <div class="copy copy-2"> <div class="container-fluid"> <div class="row"> <div class="offset-md-2 col-md-10 offset-lg-5 col-lg-7"> <p class="head-text"><span class="font-bold">ONPATTRO<sup>®</sup> (patisiran)</span> can reverse polyneuropathy manifestations of the disease<sup>4,5</sup> </p> </div> <div class="offset-md-3 col-md-9 offset-lg-6 col-lg-6"> <div class="subheader-content-wrapper"> <p class="subhead-text font-bold">ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.</p> <p class="subhead-text font-bold">At 18 months in a placebo-controlled study, ONPATTRO demonstrated<sup>4</sup>:</p> <ul class="leaf-purple-list"> <li>Reversal in neuropathy impairment from baseline as measured by modified Neuropathy Impairment Score + 7 (mNIS+7) </li> <li>Improvement in quality of life from baseline as measured by Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) score </li> </ul> <button type="button" class="btn btn-primary link-container" id="efficacy-data"><a href="/efficacy">See efficacy data</a> </button> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="page-content-section"> <div class="container"> <div class="row"> <div class="col-md-12"> <div class="cards-container"><a href="/dosing-administration" class="custom-card" id="first"> <img alt="Icon left arrow" src="/themes/onp/img/icon-navigation-arrow-teal.svg" class="teal-arrow blue"> <img alt="Icon left arrow" src="/themes/onp/img/icon-navigation-arrow-teal-green.svg" class="teal-arrow green"> <div class="icon-container my-auto"><img alt="Infusion bag" src="/themes/onp/img/dosing-administration.svg"></div> <div class="content-container"> <div class="content-header font-bold"> <span>Learn more about dosing<br> and administration</span> </div> <div class="card-content">See step-by-step instructions on how to prepare and administer ONPATTRO</div> </div> </a> <a href="/start-form" class="custom-card" id="second"> <img alt="Icon left arrow" src="/themes/onp/img/icon-navigation-arrow-teal.svg" class="teal-arrow blue"> <img alt="Icon left arrow" src="/themes/onp/img/icon-navigation-arrow-teal-green.svg" class="teal-arrow green"> <div class="icon-container my-auto"><img alt="DNA helix" src="/themes/onp/img/get-started.svg"></div> <div class="content-container"> <div class="content-header font-bold"> <span>Get started with<br> ONPATTRO </span> </div> <div class="card-content">Find out how to start your<br> patients on ONPATTRO</div> </div> </a> <a href="/alnylam-assist" class="custom-card" id="third"> <img alt="Icon left arrow" src="/themes/onp/img/icon-navigation-arrow-teal.svg" class="teal-arrow blue"> <img alt="Icon left arrow" src="/themes/onp/img/icon-navigation-arrow-teal-green.svg" class="teal-arrow green"> <div class="icon-container my-auto"><img alt="Case Manager" src="/themes/onp/img/patients-support.svg"></div> <div class="content-container"> <div class="content-header font-bold"> <span>Support for your<br> patients</span> </div> <div class="card-content">Discover how Alnylam Assist<sup>®</sup><br> can help support patients</div> </div> </a> </div> </div> <div class="col-md-12"> <div class="stay-informed-section"> <div class="row"> <div class="col-md-7 offset-md-1"> <p class="section-header font-bold">Stay informed</p> <p class="section-subheader">Receive news and updates about ONPATTRO</p> </div> <div class="col-md-3 d-flex justify-content-end"> <button type="button" class="btn btn-primary link-container"><a href="/sign-up">Sign up now</a> </button> </div> </div> </div> </div> <div id="isi"> <div class="row" id="isi-content"> <div class="col-12"> <p class="header font-bold">Important Safety Information and Indication</p> <p class="subheader font-bold">Important Safety Information</p> <p class="section-header font-bold">Infusion-Related Reactions</p> <p>Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO<sup>®</sup> (patisiran). In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.</p> <p>To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.</p> <p class="section-header font-bold">Reduced Serum Vitamin A Levels and Recommended Supplementation</p> <p>ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.</p> <p>Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).</p> <p class="section-header font-bold">Adverse Reactions</p> <p>The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion-related reactions (19%).</p> <p class="subheader last-section font-bold">Indication</p> <p>ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.</p> <p class="section-header font-bold">For additional information about ONPATTRO, please see the full <a href="https://alnylam.com/onpattro-us-prescribing-information" target="_blank" class="prescribing-information">Prescribing Information</a>.</p> </div> </div> </div> <div class="col-12 padding-0" id="references"> <div class="references-container"> <p class="references-header">References:</p> <p class="reference-text"><span class="reference-number">1.</span> Ando Y, Coelho T, Berk JL, et al. <em>Orphanet J Rare Dis.</em> 2013;8:31.</p> <p class="reference-text"><span class="reference-number">2.</span> Coutinho P, Martins da Silva A, Lopes Lima JL, et al. Excerpta Medica; 1980:88-98.</p> <p class="reference-text"><span class="reference-number">3.</span> Vinik EJ, Vinik AI, Paulson JF, et al. <em>J Peripher Nerv Syst.</em> 2014;19:104-119.</p> <p class="reference-text"><span class="reference-number">4.</span> ONPATTRO Prescribing Information. Cambridge, MA: Alnylam Pharmaceuticals, Inc.</p> <p class="reference-text"><span class="reference-number">5.</span> Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. <em>N Engl J Med.</em> 2018;379(1):11-21.</p> </div> </div> </div> </div> </div> </div> <div class="isi-floating-content" id="isi-floating-content"> <div class="container"> <div class="row"> <div class="col-12"><img class="open-close-icon rotate--90" src="/themes/onp/img/icon-navigation-arrow-teal.svg"></div> </div> <div class="row" id="isi-content"> <div class="col-12"> <p class="header font-bold">Important Safety Information and Indication</p> <p class="subheader font-bold">Important Safety Information</p> <p class="section-header font-bold">Infusion-Related Reactions</p> <p>Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO<sup>®</sup> (patisiran). In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.</p> <p>To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.</p> <p class="section-header font-bold">Reduced Serum Vitamin A Levels and Recommended Supplementation</p> <p>ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.</p> <p>Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).</p> <p class="section-header font-bold">Adverse Reactions</p> <p>The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion-related reactions (19%).</p> <p class="subheader last-section font-bold">Indication</p> <p>ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.</p> <p class="section-header font-bold">For additional information about ONPATTRO, please see the full <a href="https://alnylam.com/onpattro-us-prescribing-information" target="_blank" class="prescribing-information">Prescribing Information</a>.</p> </div> </div> </div> </div> <footer class="footer"> <div class="container"> <div class="row"> <div class="col-md-12"> <ul class="footer-links-container d-none d-sm-none d-md-flex"> <li class="footer-item"><a class="external-anylam-link footer-link" href="https://www.alnylampolicies.com/privacy">Privacy Policy</a></li> <li class="footer-item"><a class="external-anylam-link footer-link" href="https://www.alnylampolicies.com/legal">Legal Notice</a></li> <li class="footer-item"><a class="external-anylam-link footer-link" href="https://www.alnylam.com/contact-us">Contact Us</a></li> <li class="footer-item"><a class="footer-link" href="/site-map">Site Map</a></li> </ul> <ul class="footer-links-container d-sm-flex d-md-none" style="color: blue;"> <li class="footer-item"><a class="external-anylam-link footer-link" href="https://www.alnylampolicies.com/privacy">Privacy Policy</a></li> <li class="footer-item"><a class="external-anylam-link footer-link" href="https://www.alnylampolicies.com/legal">Legal Notice</a></li> </ul> <ul class="footer-links-container d-sm-flex d-md-none" style="color: blue;"> <li class="footer-item"><a class="external-anylam-link footer-link" href="https://www.alnylam.com/contact-us">Contact Us</a></li> <li class="footer-item"><a class="footer-link" href="/site-map">Site Map</a></li> </ul> <a href="https://www.alnylam.com" class="external-anylam-link"><img class="gilead-logo" alt="Logo Alnylam" src="/themes/onp/img/logo-alnylam.svg"></a> <p class="text-color-white m-0">ONPATTRO and Alnylam Assist are registered trademarks of Alnylam Pharmaceuticals, Inc. <br class="d-block d-md-none">© 2022 Alnylam Pharmaceuticals, Inc. 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TTR02-USA-00030-092018</p> </div> </div> </div> </footer> <div class="modal fade video-modal" id="expertPerspectivesVideo1" tabindex="-1" role="dialog" aria-labelledby="contactUsTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-lg" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <iframe id="expertPerspectivesVideo-iframe" src="https://player.vimeo.com/video/316627755" width="100%" frameborder="0" allow="autoplay; fullscreen" allowfullscreen></iframe> </div> </div> </div> </div> <div class="modal fade video-modal" id="expertPerspectivesVideo2" tabindex="-1" role="dialog" aria-labelledby="contactUsTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-lg" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <iframe id="expertPerspectivesVideo-iframe" src="https://player.vimeo.com/video/583820909" width="100%" frameborder="0" allow="autoplay; fullscreen; picture-in-picture" allowfullscreen></iframe> </div> </div> </div> </div> <div class="modal fade video-modal" id="expertPerspectivesVideo3" tabindex="-1" role="dialog" aria-labelledby="contactUsTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-lg" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <iframe id="expertPerspectivesVideo-iframe" src="https://player.vimeo.com/video/289146864" width="100%" frameborder="0" allow="autoplay; fullscreen" allowfullscreen></iframe> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="healthcareProfessionalModal" data-backdrop="static" tabindex="-1" role="dialog" aria-labelledby="healthcareProfessionalTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-lg" role="document"> <div class="modal-content"> <div class="modal-body"> <p class="font-bold font-purple text-center header">The site you are about to enter is intended for US healthcare professionals. Please confirm that you are a US healthcare professional.</p> <div class="buttons-container text-center"> <button type="button" class="btn btn-primary" data-dismiss="modal" id="stay"><span class="font-bold">I am a</span><br> healthcare professional</button> <button type="button" class="btn btn-primary" id="leave"><a href="https://www.onpattro.com"><span class="font-bold">I am not a</span><br> healthcare professional</a></button> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="primaryEndpointModal" tabindex="-1" role="dialog" aria-labelledby="primaryEndpointTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-5 offset-lg-1"> <p class="header">Primary Endpoint<sup>1,2</sup></p> <span class="font-bold font-purple">modified Neuropathy Impairment Score + 7 (mNIS+7)</span> <ul class="primary-endpoint-list"> <li><span>Validated composite measure of sensory, motor, and autonomic neuropathy</span></li> <li><span>Score ranges from 0 (no impairment) to 304 points (maximal impairment)</span></li> </ul> </div> <div class="col-lg-5"> <div class="img-container"> <img src="/themes/onp/img/graphic-m-nis-chart.svg" alt="mNIS+7 components"> </div> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="norfolkModal" tabindex="-1" role="dialog" aria-labelledby="norfolkEndpointTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-5 offset-lg-1"> <p class="header">Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN)<sup>1,8,16</sup></p> </div> <div class="col-lg-6"></div> <div class="col-lg-5 offset-lg-1"> <ul class="primary-endpoint-list"> <li> <span>Patient questionnaire designed to capture the impact of hATTR amyloidosis on a patient’s quality of life relating to: </span> <ul class="internal-list"> <li><span><span class="left-hang">—</span>Activities of daily living</span></li> <li><span><span class="left-hang">—</span>Polyneuropathy symptoms</span></li> <li><span><span class="left-hang">—</span>Autonomic function</span></li> <li><span><span class="left-hang">—</span>Small fiber nerve function</span></li> <li><span><span class="left-hang">—</span>Physical functioning/large fiber nerve function</span></li> </ul> </li> <li><span>Score ranges from -4 to 136; higher score indicates worsening quality of life</span></li> </ul> </div> <div class="col-lg-5"> <span class="font-bold font-purple">Sample questions from the questionnaire<sup>16</sup></span> <ul class="primary-endpoint-list"> <li><span>Have you felt unsteady on your feet?</span></li> <li><span>Have you had a problem walking down stairs?</span></li> <li><span>Have you had a problem with bathing or showering?</span></li> <li><span>Have you had a problem with dressing?</span></li> <li><span>Were you limited in the kind of work or other activities you could perform?</span></li> </ul> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="compass31Modal" tabindex="-1" role="dialog" aria-labelledby="compass31Title" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-5 offset-lg-1"> <p class="header">Composite Autonomic Symptom Score 31 (COMPASS 31)<sup>7,9</sup></p> </div> <div class="col-lg-6"></div> <div class="col-lg-5 offset-lg-1"> <ul class="primary-endpoint-list"> <li><span>31-item questionnaire that evaluated patient-reported autonomic symptoms such as orthostatic hypotension, constipation, diarrhea, nausea/vomiting, and incontinence</span></li> <li><span>Score ranges from 0 to 100; lower score indicates fewer autonomic symptoms</span></li> <li><span>Covers 6 autonomic domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor)<sup>2</sup></span></li> </ul> </div> <div class="col-lg-5"> <span class="font-bold font-purple">Sample questions from the questionnaire<sup>9</sup></span> <ul class="primary-endpoint-list"> <li><span>In the past year, have you ever felt faint, dizzy, “goofy,” or had difficulty thinking soon after standing up from a sitting or lying position?</span></li> <li><span>In the past year, have you ever noticed color changes in your skin, such as red, white, or purple?</span></li> <li><span>In the past 5 years, what changes, if any, have occurred in your general body sweating?</span></li> <li><span>In the past year, have you had any bouts of diarrhea?</span></li> <li><span>In the past year, have you ever lost control of your bladder function?</span></li> </ul> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="rodsModal" tabindex="-1" role="dialog" aria-labelledby="rodsTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-5 offset-lg-1"> <p class="header">Rasch-built Overall Disability Scale (R-ODS)<sup>7,11,17</sup></p> </div> <div class="col-lg-6"></div> <div class="col-lg-5 offset-lg-1"> <ul class="primary-endpoint-list"> <li><span>24-item scale that evaluated patient-reported ability to perform activities of daily living such as eating, bathing, dressing, and standing</span></li> <li><span>Score ranges from 0 to 48; higher score indicates less disability</span></li> </ul> </div> <div class="col-lg-5"> <span class="font-bold font-purple">Sample questions from the evaluation include<sup>11,17</sup></span> <ul class="primary-endpoint-list"> <li> <span>Are you able to:</span> <ul class="internal-list"> <li><span><span class="left-hang">—</span>Walk 1 flight of stairs?</span></li> <li><span><span class="left-hang">—</span>Take a shower?</span></li> <li><span><span class="left-hang">—</span>Carry and put down a heavy object?</span></li> <li><span><span class="left-hang">—</span>Do the shopping?</span></li> </ul> </li> </ul> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="walkingSpeedModal" tabindex="-1" role="dialog" aria-labelledby="walkingSpeedTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-10 offset-lg-1"> <p class="header">Walking speed<sup>1,7</sup></p> <ul class="primary-endpoint-list"> <li><span>Gait speed measured in meters per second (m/sec) using 10-meter walk test (10MWT)</span></li> <li><span>A higher number indicates less disability/less impairment</span></li> </ul> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="nutritionalStatusModal" tabindex="-1" role="dialog" aria-labelledby="nutritionalStatusTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-10 offset-lg-1"> <p class="header">Nutritional status<sup>1,18</sup></p> <ul class="primary-endpoint-list"> <li><span>Nutritional status assessment based on body mass index and serum albumin (kg/m<sup>2</sup> x albumin [g/L])</span></li> <li><span>Lower value indicates worsening of nutritional status</span></li> <li><span>mBMI is used as a tool to monitor disease progression and provide prognostic information, showing close correlation with time before death, duration of gastrointestinal disturbances, malabsorption, and functional capacity</span></li> </ul> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="motorStrengthModal" tabindex="-1" role="dialog" aria-labelledby="motorStrengthTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-10 offset-lg-1"> <p class="header">Motor strength<sup>2,7</sup></p> <ul class="primary-endpoint-list"> <li><span>Motor strength measured on the weakness component of the Neuropathy Impairment Score (NIS-W)</span></li> <li><span>Score ranges from 0 to 192; higher score indicates more impairment</span></li> </ul> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="patientMobilityModal" tabindex="-1" role="dialog" aria-labelledby="patientMobilityTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-xl" role="document"> <div class="modal-content"> <div class="modal-header"> <button type="button" class="close" data-dismiss="modal" aria-label="Close"> <img src="/themes/onp/img/icon-navigation-close-teal.svg"> </button> </div> <div class="modal-body"> <div class="row"> <div class="col-lg-5 offset-lg-1"> <p class="header">EuroQoL 5 Dimensions 5 Levels (EQ-5D-5L)<sup>14</sup></p> </div> <div class="col-lg-6"></div> <div class="col-lg-5 offset-lg-1"> <ul class="primary-endpoint-list"> <li> <span>Instrument used to measure health in 5 dimensions: </span> <ul class="internal-list"> <li><span><span class="left-hang">—</span>Mobility (walking)</span></li> <li><span><span class="left-hang">—</span>Self-care (washing or dressing)</span></li> <li><span><span class="left-hang">—</span>Usual activities</span></li> <li><span><span class="left-hang">—</span>Pain/discomfort</span></li> <li><span><span class="left-hang">—</span>Anxiety/depression</span></li> </ul> </li> <li><span>Score ranges from 0 to 1; lower score indicates worsening quality of life</span></li> </ul> </div> <div class="col-lg-5"> <span class="font-bold font-purple">Example:</span> <ul class="primary-endpoint-list"> <li> <span>Anxiety/Depression</span> <ul class="green-square"> <li><span>I am not anxious or depressed</span></li> <li><span>I am slightly anxious or depressed</span></li> <li><span>I am moderately anxious or depressed</span></li> <li><span>I am severely anxious or depressed</span></li> <li><span>I am extremely anxious or depressed</span></li> </ul> </li> </ul> <p class="under-list-text">EQ-5D-5L was an exploratory endpoint in the APOLLO study.</p> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="emailAlreadyInUseModal" tabindex="-1" role="dialog" aria-labelledby="emailAlreadyInUseTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-lg" role="document"> <div class="modal-content"> <div class="modal-body"> <div class="row"> <div class="col-lg-12 text-center"> <img class="modal-action-id" src="/themes/onp/img/exclamation.svg" alt=""> </div> <div class="col-lg-12"> <p class="header">This email address is already in use.</p> <p class="disclaimer">Please press Cancel to enter another email, or press Update to receive an email that will allow you to update your preferences.</p> </div> <div class="col-lg-12 text-center"> <div class="actions-container"> <button type="button" class="btn btn-primary" data-dismiss="modal" id="cancel">Cancel</button> <button type="button" class="btn btn-primary" id="Update">Update</button> </div> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="emailSentModal" tabindex="-1" role="dialog" aria-labelledby="emailSentTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-lg" role="document"> <div class="modal-content"> <div class="modal-body"> <div class="row"> <div class="col-lg-12 text-center"> <img class="modal-action-id" src="/themes/onp/img/icon-checkmark-email-sent.svg" alt=""> </div> <div class="col-lg-12"> <p class="header">Email sent!</p> <p class="disclaimer">A link has been sent to your email address.</p> </div> <div class="col-lg-12 text-center"> <div class="actions-container"> <button type="button" class="btn btn-primary" data-dismiss="modal" id="ok">Ok</button> </div> </div> </div> </div> </div> </div> </div> <div class="modal fade endpoint-modal" id="nonAnylamLinkModal" tabindex="-1" role="dialog" aria-labelledby="nonAnylamLinkTitle" aria-hidden="true"> <div class="modal-dialog modal-dialog-centered modal-lg" role="document"> <div class="modal-content"> <div class="modal-body"> <div class="row"> <div class="col-lg-12"> <p class="header">You are now leaving onpattrohcp.com</p> <p class="disclaimer">The following content may not be associated with Alnylam Pharmaceuticals. 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