Today, the Myotonic Dystrophy Foundation stands firmly with the entire myotonic dystrophy community. The news that the Phase 3 HARBOR trial of del-desiran did not meet its primary endpoint (vHOT: video hand opening time, a measure of hand myotonia) is deeply disappointing, particularly for the people living with DM1 who participated in the trial, the families who supported them, and everyone who has placed hope in the possibility of a new treatment.<\/p>\n
We recognize how deeply this news may affect our community. We know the courage it took for our community to participate in HARBOR, the commitment of their families, and the hope that accompanied every step of the trial. Today, we stand with every person and family who is processing this news. We are here, we are listening, and we will continue to walk alongside the community as we learn more.<\/strong><\/p>\n At the same time,\u00a0this result is not the end of the story.<\/strong>\u00a0A clinical trial generates important information even when it does not achieve its primary endpoint. The HARBOR study has given researchers new data about DM1, about del-desiran, and about how we evaluate potential treatments for this complex disease. That knowledge matters. It adds to our understanding of DM1 and can help guide the next steps in the search for effective treatments.<\/p>\n Importantly,\u00a0there is still additional HARBOR data to be reviewed and understood.<\/strong> The primary endpoint is one part of a much larger body of information collected during the trial. MDF will continue to engage with Novartis as additional results and analyses become available, and we will work to ensure that the community receives clear, timely, and understandable information about what the full dataset tells us. Read Novartis’s statement to the DM community,<\/a> which references their original announcement<\/a> from earlier today.<\/p>\n \u201cThere are no words that can erase the disappointment our community is feeling today,\u201d said Dr. Tanya Stevenson, CEO of the Myotonic Dystrophy Foundation. \u201cWe are thinking especially of the people who took the courageous step of joining this trial. Their contribution matters. Their voices matter. And their hope for a better future matters. The information generated through HARBOR will continue to be studied, and that knowledge will help move DM research forward. We owe it to every patient and family affected by DM1 to keep pursuing the answers, to learn from every study, and to keep moving the field forward. There is reason to remain hopeful about what we can accomplish together.\u201d<\/p>\n Jeremy Kelly, Chair of the MDF Board of Directors, added, \u201cWe recognize the profound disappointment that today\u2019s news brings. Myotonic dystrophy affects every part of life for children, parents, spouses, and entire families. People living with DM have waited long enough for treatments that make a meaningful difference. Today\u2019s news is a setback, but it does not change the urgency of that need or the determination of the research community and MDF to meet it. We will learn from these results, we will keep asking questions, and we will keep moving forward.\u201d<\/p>\n Every clinical trial teaches us something.<\/strong>\u00a0Sometimes the path to a treatment is direct; more often, it is built through one piece of knowledge at a time. The HARBOR results are another piece of that knowledge. As researchers continue to examine the full dataset, we will learn more about what the results mean and what questions they raise for the future of DM1 treatment development.<\/p>\n MDF remains unwavering in its commitment to the people and families living with myotonic dystrophy. We will continue to advocate for rigorous research, support patients through every stage of their journey, and work alongside researchers, companies, regulators, and the broader DM community to accelerate the development of treatments that can meaningfully improve the lives of people living with DM.<\/p>\n There is still work to do. There are still questions to answer. There are still many ongoing trials to learn from, including HARBOR – and there is still a future to build.<\/strong><\/p>\n To everyone in the DM community: we see you, we hear you, and we are standing with you. The road may be difficult, but\u00a0we remain steadfast in our commitment to a future with meaningful treatments for myotonic dystrophy.<\/p>\n","protected":false},"excerpt":{"rendered":" Today, the Myotonic Dystrophy Foundation stands firmly with the entire myotonic dystrophy community. The news that the Phase 3 HARBOR trial of del-desiran did not meet its primary endpoint (vHOT: video hand opening time, a measure of hand myotonia) is deeply disappointing, particularly for the people living with DM1 who participated in the trial, the […]<\/p>\n","protected":false},"author":10,"featured_media":6011,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[161,192],"tags":[],"class_list":["post-27404","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-non-classe","category-recherche"],"_links":{"self":[{"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/posts\/27404","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/users\/10"}],"replies":[{"embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/comments?post=27404"}],"version-history":[{"count":6,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/posts\/27404\/revisions"}],"predecessor-version":[{"id":27410,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/posts\/27404\/revisions\/27410"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/media\/6011"}],"wp:attachment":[{"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/media?parent=27404"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/categories?post=27404"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/tags?post=27404"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}