{"id":12188,"date":"2025-02-28T11:37:45","date_gmt":"2025-02-28T19:37:45","guid":{"rendered":"https:\/\/mr66givry2.onrocket.site\/?p=12188"},"modified":"2026-02-19T14:29:01","modified_gmt":"2026-02-19T22:29:01","slug":"global-alliance-60-myotonic-dystrophy-organizations-unites-rare-disease-day-2025-2","status":"publish","type":"post","link":"https:\/\/myotonic.org\/fr\/global-alliance-60-myotonic-dystrophy-organizations-unites-rare-disease-day-2025-2\/","title":{"rendered":"Global Alliance of 60+ Myotonic Dystrophy Organizations Unites for Rare Disease Day 2025"},"content":{"rendered":"
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Click here to download this press release as a printable PDF.<\/a><\/p>\n

PRESS RELEASE<\/strong><\/p>\n

Canada Contact Name: Homira Osman
\nCanada Contact Email:\u00a0Homira.osman@muscle.ca<\/a><\/p>\n

Japan Contact Name: Midori Senoo
\nJapan Contact Email:\u00a0international@dm-family.net<\/a><\/p>\n

UK Contact Names: Stephen Uncles
\nUK Contact Emails:\u00a0stephen.uncles@sky.com<\/a><\/p>\n

US Contact Name: Tanya Stevenson
\nUS Contact Email:\u00a0Tanya.Stevenson@myotonic.org<\/a><\/p>\n

FOR IMMEDIATE RELEASE<\/strong><\/p>\n

Global Alliance of 60+ Myotonic Dystrophy Organizations Unites for Rare Disease Day 2025<\/h2>\n

February 28, 2025, Worldwide<\/strong>\u00a0\u2013 The\u00a0Global Alliance for Myotonic Dystrophy Awareness<\/a>\u00a0(Global Alliance) proudly joins the global rare disease community in commemorating Rare Disease Day on February 28th, 2025. With over 60 organizations collaborating worldwide, the Alliance continues to advance understanding, care, and research for myotonic dystrophy (DM), one of the most prevalent rare genetic disorders.<\/p>\n

Myotonic dystrophy (DM)<\/a>\u00a0is a progressive, inherited disorder that impacts muscle strength, respiratory systems, heart function, and cognitive abilities, among other areas. Despite its prevalence, DM remains underdiagnosed and misunderstood, with no current cure or disease-modifying treatments available. The Global Alliance is committed to addressing these gaps through collaboration and education.<\/p>\n

In 2025, the Global Alliance reaffirms its commitment to two joint areas of focus:<\/p>\n

    \n
  1. Raising awareness and providing education among clinical care teams.<\/strong><\/li>\n
  2. Enhancing clinical trial readiness for participants.<\/strong><\/li>\n<\/ol>\n

    These priorities address the critical challenges faced by individuals and families affected by myotonic dystrophy. In line with its renewed priorities, the Global Alliance will continue working with healthcare providers to enhance awareness and understanding of DM, its symptoms, and\u00a0available care guidelines<\/a>. The aim of this education is to\u00a0reduce delays in diagnosis<\/a>\u00a0and improve the quality of care provided to individuals affected by the disease.<\/p>\n

    Muscular Dystrophy Canada, member of the Global Alliance, recently launched\u00a0an initiative to improve access to genetic testing<\/a>\u00a0for individuals impacted by myotonic dystrophy. This groundbreaking effort aims to address one of the key barriers to timely diagnosis and participation in research. \u00ab\u00a0Genetic testing is the cornerstone of understanding and managing myotonic dystrophy, and improving access will empower individuals and families with critical information,\u00a0\u00bb said Dr. Homira Osman, Vice-President of Research and Public Policy at\u00a0Muscular Dystrophy Canada<\/a>. \u00ab\u00a0By ensuring equitable access to testing, we can pave the way for earlier diagnoses, better care, and greater inclusion in clinical trials. Together, we\u2019re making strides toward meaningful change for the DM community.\u201d Muscular Dystrophy Canada\u2019s new initiative embodies the Global Alliance\u2019s shared areas of focus by increasing awareness among clinical care teams while also helping those living with DM to get necessary information about clinical trials.<\/p>\n

    The Global Alliance continues work to prepare participants around the globe for clinical trials, which are crucial research studies testing treatments and therapies for myotonic dystrophy. The Global Alliance is dedicated to improving access to trial information and providing support for those interested in participating, while also advocating for inclusive and accessible trial designs that accommodate the diverse needs of the DM community.<\/p>\n

    \u00ab\u00a0Myotonic dystrophy registries play an essential role in bridging the gap between patients and\u00a0clinical trials<\/a>,\u00a0\u00bb said Helen Walker, Registry Curator for the\u00a0UK DM Patient Registry<\/a>. \u00ab\u00a0By collecting and maintaining accurate data shared by patients, registries not only help researchers understand the disease better but also ensure that patients are aware of and prepared for opportunities to participate in trials and other research opportunities. This is a vital step toward developing effective treatments and improving quality of life for the DM community.\u00a0\u00bb<\/p>\n

    Dr. Andy Berglund, Director of the\u00a0RNA Institute at the University at Albany<\/a>, highlighted the growing momentum in DM research: \u00ab\u00a0The strides being made in myotonic dystrophy research would not be possible without the collective efforts of patients, clinicians, and advocacy groups. The renewed focus on clinical trial readiness ensures that the DM community is well-positioned to benefit from emerging therapies. At the RNA Institute, we are honored to be part of the Global Alliance to drive progress and create hope for those living with DM.\u00a0\u00bb<\/p>\n

    The Global Alliance invites everyone\u2014individuals living with DM, families, healthcare professionals, industry, and researchers\u2014to join in raising awareness and advocating for those living with myotonic dystrophy on Rare Disease Day. This day provides a unique opportunity to unite and amplify the voices of those impacted by DM. Activities can include sharing personal stories with loved ones, engaging policymakers to\u00a0advocate for research funding and improved care<\/a>, providing clinical\u00a0care guidelines<\/a>\u00a0to healthcare providers, or even\u00a0hosting fundraising events<\/a>.<\/p>\n

    By working together, we can build a brighter future for the DM community.<\/strong><\/p>\n

    For more information, visit the\u00a0Global Alliance for Myotonic Dystrophy Awareness<\/a>.<\/p>\n

    ###<\/strong><\/p>\n

     <\/p>\n

    The Global\u00a0Alliance for Myotonic Dystrophy Awareness<\/h2>\n

    Members listed alphabetically by country. An * indicates a founding member of the Myotonic Dystrophy Awareness Global Alliance.<\/p>\n

    \n
    \n
    \n

    <\/a><\/p>\n

    Alg\u00e9ria<\/strong>\u00a0–\u00a0Association Shifa of Neuromuscular Diseases (ASMNM)<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Australia<\/strong>\u00a0–\u00a0MDA Australia<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Belgium<\/strong>\u00a0–\u00a0Association Belge contre les Maladies neuro-Musculaires<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Canada<\/strong>\u00a0–\u00a0The Neuromuscular Disease Network for Canada<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Canada<\/strong>\u00a0–\u00a0Muscular Dystrophy Canada<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Denmark<\/strong>\u00a0–\u00a0Muskelsvindfonden<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Europe<\/strong>\u00a0–\u00a0Euro-DyMA<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    France<\/strong>\u00a0–\u00a0AFM-T\u00e9l\u00e9thon<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Germany<\/strong>\u00a0–\u00a0Deutschen Gesellschaft f\u00fcr Muskelkranke e.V.<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Greece<\/strong>\u00a0–\u00a0Muscular Dystrophy Association HELLAS<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Italy<\/strong>\u00a0–\u00a0Centro Clinico NeMO<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Italy<\/strong>\u00a0–\u00a0FMM – Fondazione Malattie Miotoniche<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Japan<\/strong>\u00a0–\u00a0Myotonic Dystrophy Patients’ Group of Japan<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Netherlands<\/strong>\u00a0–\u00a0Maastricht UMC+<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Netherlands<\/strong>\u00a0–\u00a0Stichting MD (Nederland)<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Netherlands<\/strong>\u00a0–\u00a0Spierziekten Nederland<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    New Zealand<\/strong>\u00a0–\u00a0Centre for Brain Research, Neurogenetic Clinic, University of Auckland<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Serbia<\/strong>\u00a0–\u00a0University of Belgrade – Faculty of Biology, Center for Human Molecular Genetics<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Spain<\/strong>\u00a0–\u00a0Arthex Biotech<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Spain<\/strong>\u00a0–\u00a0CIBER<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Spain<\/strong>\u00a0–\u00a0INCLIVA. Health Research Institute<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Spain<\/strong>\u00a0–\u00a0Universitat de Val\u00e8ncia<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Switzerland<\/strong>\u00a0–\u00a0ASRIMM (Association Suisse Romande Intervenant contre les Maladies neuro-Musculaires)<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Switzerland<\/strong>\u00a0–\u00a0Lupin Neurosciences<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0AMO Pharma Ltd<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0Cure DM Myotonic Dystrophy UK<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0Myotonic Dystrophy Support Group<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0Muscular Dystrophy UK<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0St George’s University Hospitals NHS Foundation Trust<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0TREAT-NMD<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0UK Myotonic Dystrophy Patient Registry<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    United Kingdom<\/strong>\u00a0–\u00a0University College London Hospitals NHS Foundation Trust<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0AskBio<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Astellas Gene Therapies<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Athens Academy<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Avidity Biosciences<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Dyne Therapeutics<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Entrada Therapeutics<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Expansion Therapeutics<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0GrittGene Therapeutics<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Harmony Biosciences<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Houston Methodist Department of Neurology<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Juvena Therapeutics, Inc.<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Kansas State University Wildcat Extension<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Indiana University School of Medicine, Dept of Neurology<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0The Jackson Laboratory (JAX)<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Muscular Dystrophy Association USA<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Myotonic Dystrophy Foundation<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Myotonic Dystrophy Family Registry<\/a>*<\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0National Registry for Myotonic Dystrophy (DM) & Facioscapulohumeral Dystrophy (FSHD)\u00a0<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Paul D. Wellstone Muscular Dystrophy Cooperative Research Center<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0PepGen<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Stanford University<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0University of Florida Center for Neurogenetics<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0NeuroGenetics Student Organization at the University of Florida<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0The RNA Institute, University at Albany, SUNY\u00a0<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0University of Iowa Health Care\u00a0<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0University of Utah<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Vertex Pharmaceuticals<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Virginia Commonwealth University<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    USA<\/strong>\u00a0–\u00a0Wake Forest University, Department of Neurology<\/a><\/p>\n<\/div>\n

    \n

    <\/a><\/p>\n

    Worldwide<\/strong>\u00a0–\u00a0International Myotonic Dystrophy Consortium<\/a>*<\/p>\n<\/div>\n<\/div>\n<\/div>\n

    An * indicates a founding member of the Myotonic Dystrophy Awareness Global Alliance.<\/p>\n

    If you are a group, organization, hospital, academic institution or company interested in officially joining the Global Alliance to raise awareness of myotonic dystrophy and would like to add your name and logo to the team,\u00a0please complete this form.<\/a><\/p>\n<\/div>\n<\/div>\n","protected":false},"excerpt":{"rendered":"

    Click here to download this press release as a printable PDF. PRESS RELEASE Canada Contact Name: Homira Osman Canada Contact Email:\u00a0Homira.osman@muscle.ca Japan Contact Name: Midori Senoo Japan Contact Email:\u00a0international@dm-family.net UK Contact Names: Stephen Uncles UK Contact Emails:\u00a0stephen.uncles@sky.com US Contact Name: Tanya Stevenson US Contact Email:\u00a0Tanya.Stevenson@myotonic.org FOR IMMEDIATE RELEASE Global Alliance of 60+ Myotonic Dystrophy Organizations […]<\/p>\n","protected":false},"author":9,"featured_media":12189,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[70],"tags":[],"class_list":["post-12188","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"_links":{"self":[{"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/posts\/12188","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\/9"}],"replies":[{"embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/comments?post=12188"}],"version-history":[{"count":0,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/posts\/12188\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/media\/12189"}],"wp:attachment":[{"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/media?parent=12188"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/categories?post=12188"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/myotonic.org\/fr\/wp-json\/wp\/v2\/tags?post=12188"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}