{"id":6874,"date":"2026-06-04T18:26:51","date_gmt":"2026-06-04T18:26:51","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=6874"},"modified":"2026-06-08T14:24:10","modified_gmt":"2026-06-08T14:24:10","slug":"idiopathic-short-stature-iss","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/idiopathic-short-stature-iss\/","title":{"rendered":"Idiopathic Short Stature (ISS)"},"content":{"rendered":"<div id=\"acf-block-68de3cac2ef74\" class=\"hero hero-no-overlay hero-large-first-para\">\n            <div class=\"hero-background-image hero-background-image-desktop\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-bones.png?v=1.22);\"><\/div>\n\t    <div class=\"hero-background-image hero-background-image-mobile\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-bones-Mobile.png?v=1.22);\"><\/div>\n    \t<div class=\"overlay\"><\/div>\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"hero-content\">\n\t\t\t    \t\t\t\t\t\t\t\t\t\t\t\t    <h1>Idiopathic Short Stature\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>Learn more about ISS and BioMarin research<\/p>\n\t\t\t\t\t\t\t\t\n                <p class=\"inline-buttons\">\n                                                        <\/p>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n\n<div id=\"acf-block-68e7d6279a7e3\" class=\"block cards cards-2-col\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"cards-title\">\n\t\t\t    \t\t\t\t\t\t\t<\/div>\n\t\t\t<div class=\"cards-wrapper\">\n\t\t\t    \n<div id=\"acf-block-68e7d6279ad98\" class=\"card card-product card-fixed-button\" data-muted-autoplay=\"false\">\n        <div class=\"card-content\">\n                            <h4>Clinical Program\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                                                        <\/div>\n                                <div class=\"card-actions\">\n                            \n                            <p><a class=\"button button-arrow\" href=\"#acf-block-68ee5c07eba7b\" target=\"_self\">See clinical trials<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-68e7d6279ae37\" class=\"card card-product card-fixed-button\" data-muted-autoplay=\"false\">\n        <div class=\"card-content\">\n                            <h4>Congress Posters and Presentations\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                                                        <\/div>\n                                <div class=\"card-actions\">\n                            \n                            <p><a class=\"button button-arrow\" href=\"#acf-block-68ecfd62c135f\" target=\"_self\">See posters &amp; presentations<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-68ee5c07eba7b\" class=\"vblock pipeline pipeline-listing-wrapper no-pipeline-filters\">\n    <!-- Pipeline Filter Header\n    Integration notes:\n    - Toggle button-filter-active class to filter button when active\n    - Component here is generic filter header and is also used for resources (filters themselves differ)\n    -->\n    <div class=\"block filter-header\">\n        <div class=\"wrapper\">\n            <div class=\"inner-wrapper\">\n                <h1>Idiopathic Short Stature Pipeline<\/h1>\n                \n                            <\/div>\n        <\/div>\n    <\/div>\n\n    <!-- Research Pipeline block -->\n    <div class=\"block bg-band pipeline-listing-main\">\n        <div class=\"wrapper\">\n            <div class=\"inner-wrapper\">\n                                                                            \n                                        <div class=\"pipeline-listing pipeline-skeletal-conditions\">\n                        <h2 class=\"h3\">Skeletal Conditions<\/h2>\n\n                                                                                                                                        <h3 class=\"h5 trial-phase trial-phase\">Trial phase<\/h3>\n\n                            <div class=\"pipeline-group\" data-trial-phase-class=\"trial-phase\">\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                            <div class=\"pipeline-headers\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">Program (Trial)<\/div>\n                                                <div class=\"pipeline-condition\">Condition<\/div>\n                                                <div class=\"pipeline-technology\">Technology<\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                        <\/div>\n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 phase-2 filter-condition-idiopathic-short-stature filter-tech-peptide filter-rec-stat-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Vosoritide<\/h3>\n                                                    <p>(111-210 [Canopy ISS-2])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Idiopathic Short Stature<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Peptide<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>This study will evaluate the effect of multiple doses of vosoritide and the effect of vosoritide compared to human growth hormone (hGH), in children with Idiopathic Short Stature.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>Change in annualized growth velocity (AGV), height, and height Z-score<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Incidence of adverse events<\/li>\n<li>Change in pharmacodynamic parameters (urine cyclic guanine monophosphate, serum collagen X marker)<\/li>\n<li>Pharmacokinetics of vosoritide<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Patients &ge;3 and &lt;10 (female) or &lt; 11 (males) years of age<\/li>\n<li>Height Z-score &#x2264;-2.25 standard deviations compared to age and sex matched population norms<\/li>\n<li>If &#x2265; 5 years at Screening, must be Tanner Stage I<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to: Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency<\/li>\n<li>History of treatment with a growth promoting agent<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06382155\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06382155<\/a><\/p>\n                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial observational filter-condition-idiopathic-short-stature filter-tech-observational filter-rec-stat-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Idiopathic Short Stature<\/h3>\n                                                    <p>(111-903 [Canopy ISS-OS])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Idiopathic Short Stature<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Observational<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>A Study to Assess Growth in Children With Idiopathic Short Stature<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>This study will collect growth measurements and other variables of interest to generate baseline growth data in children with Idiopathic Short Stature.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>Change in growth (annualized growth velocity, height Z-score, height) and anthropometric measures (body mass index [BMI], BMI Z-score)<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li class=\"ewa-rteLine\">Rates of medical events of interest<\/li>\n<li class=\"ewa-rteLine\">Association between specific variants and growth velocity<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Patients &gt; 2 and &le; 14 (female) or &le; 16 (males) years of age<\/li>\n<li>Height Z-score &#x2264;-2.5 standard deviations compared to age and sex matched population norms<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li class=\"ewa-rteLine\">Presence of pituitary hormone deficiencies<\/li>\n<li class=\"ewa-rteLine\">Bone age advanced over chronological age by &gt;3 years<\/li>\n<li class=\"ewa-rteLine\">Received an investigational product or medical device within last 6 months<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06309979\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06309979<\/a><\/p>\n                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                            <\/div>\n                                            <\/div>\n                            <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68ecfd62c135f\" class=\"block resources-listing\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\n                            <div class=\"listing-title\">\n                                            <h2>Congress Posters and Presentations\n<\/h2>\n                                                                <p style=\"margin: 0px 0px 1rem;padding: 0px;font-size: 20px;line-height: 1.25em;color: #241c15;font-family: futura-pt, sans-serif\">All displayed posters and presentations are protected by copyright and are intended for individual use only.<\/p>\n<p style=\"margin: 0px 0px 1rem;padding: 0px;font-size: 20px;line-height: 1.25em;color: #241c15;font-family: futura-pt, sans-serif\"><i><b>Please Note<\/b>: Poster content may be unavailable or delayed. This could be due to copyright or embargo guidelines.<\/i><\/p>\n<p style=\"margin: 0px 0px 1rem;padding: 0px;font-size: 20px;line-height: 1.25em;color: #241c15;font-family: futura-pt, sans-serif\">You may check back later or contact <a style=\"color: #ed037c\" href=\"mailto:medinfo@bmrn.com\">medinfo@bmrn.com<\/a> or <a class=\"external_link\" style=\"color: #ed037c\" href=\"https:\/\/www.biomarin.com\/web-request-form?_gl=1*1hs6n10*_ga*MTg0MDExMDAwMS4xNjg2MDQ0Nzc4*_ga_KGGE14VN95*MTY4NjEzNTY1Ni4xMC4xLjE2ODYxMzU3OTAuMC4wLjA.&amp;_ga=2.9066165.141314881.1686044782-1840110001.1686044778\" target=\"_blank\" rel=\"noopener\">complete a request form<\/a> to inquire about this specific material<\/p>\n                                    <\/div>\n\t\t\t\t\t\t                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/ESPE-ESE_2025-210-ISS_Study_Design-poster-MMRC-ISS-00101_MAY-2025-1.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Idiopathic Short Stature (ISS)<\/li>\n                                                                                                    <li>ESE<\/li>\n                                                                                                    <li>ESPE<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Design of a phase 2, randomized, controlled, multicentre study of vosoritide treatment in children with idiopathic short stature\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                            \t                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/ESPE-ESE_2025-210-ISS_Study_Design-poster-MMRC-ISS-00101_MAY-2025-1.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/10\/ESPE-2024_Comparison-MMRC-ACH-00401-T1.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Idiopathic Short Stature (ISS)<\/li>\n                                                                                                    <li>ESPE<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Comparison of the diagnostic yield of whole exome sequencing (WES) and targeted panel sequencing for children with idiopathic short stature (ISS)\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                            \t                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/10\/ESPE-2024_Comparison-MMRC-ACH-00401-T1.pdf?v=1.22\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                            \t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":3,"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-6874","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.7 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Idiopathic Short Stature (ISS) | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"Learn more about Duchenne muscular dystrophy (DMD) and BioMarin research\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/medical.biomarin.com\/en-us\/idiopathic-short-stature-iss\/\" \/>\n<meta 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