{"id":13228,"date":"2023-02-28T10:05:41","date_gmt":"2023-02-28T18:05:41","guid":{"rendered":"https:\/\/mr66givry2.onrocket.site\/?p=13228"},"modified":"2026-01-01T10:06:44","modified_gmt":"2026-01-01T18:06:44","slug":"global-alliance-57-myotonic-dystrophy-focused-organisations-unite-raise-awareness-rare-disease-day","status":"publish","type":"post","link":"https:\/\/myotonic.org\/de\/global-alliance-57-myotonic-dystrophy-focused-organisations-unite-raise-awareness-rare-disease-day\/","title":{"rendered":"Global Alliance of 57+ Myotonic Dystrophy Focused Organisations Unite to Raise Awareness on Rare Disease Day 2023"},"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

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

UK Contact Name: Emma-Jayne Ashley
\nUK Contact Email:\u00a0CureDM@outlook.com<\/a><\/p>\n

Netherlands Contact Name: Jorg van Gent
\nNetherlands Contact Email:\u00a0Jorg.vanGent@md.nl<\/a><\/p>\n

Japan Contact Name: Midori Senoo
\nJapan Contact Email:\u00a0Contact@dm-family.net<\/a><\/p>\n

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

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Global Alliance of 57+ Myotonic Dystrophy Focused Organisations Unite to Raise Awareness on Rare Disease Day 2023<\/h2>\n

February 28, 2023, Worldwide:<\/u>\u00a0On Rare Disease Day 2023, the\u00a0Global Alliance for Myotonic Dystrophy Awareness<\/a>\u00a0proudly stands together to celebrate the remarkable strength and resilience of the myotonic dystrophy community. With over 57 organisations focused on advancing the understanding of and care for this rare genetic disorder, the Alliance continues to be a beacon of hope for people living with myotonic dystrophy (DM), their families, and healthcare professionals around the globe.<\/p>\n

The Global Alliance for Myotonic Dystrophy Awareness invites individuals, organisations, and policymakers around the world to join us in raising awareness of myotonic dystrophy and advocating for improved care and research for those living with the disease.<\/p>\n

Myotonic dystrophy (DM) is a rare and\u00a0complex disease<\/a>\u00a0affecting muscle strength and wasting, respiratory systems, heart function, cognitive abilities, and more. DM is an inherited disease that affects successive generations and is often misdiagnosed and poorly supported.\u00a0With as many as 1 in 2,100 individuals at risk of developing the disease or passing it on to the next generation<\/a>, myotonic dystrophy is the most common form of adult-onset muscular dystrophy. Despite its prevalence and complexity, there are currently no disease-modifying treatments or cures available for this debilitating condition.<\/p>\n

While myotonic dystrophy affects people all around the world, possibly including people in your local community, it remains a relatively unknown disorder. By joining forces to raise awareness, the Global Alliance aims to shed light on the challenges faced by those living with myotonic dystrophy and to increase awareness and understanding of the disease across the globe.<\/p>\n

\u201cWith thousands of people in the US affected by this relatively unknown disease, we will continue to focus on the urgent need for improved diagnosis, treatment, and research to better serve the myotonic dystrophy community,\u201d said Dr. Sharon Hesterlee, Chief Research Officer at the\u00a0Muscular Dystrophy Association<\/a>. „By working together, we can improve the lives of those affected by DM and create a more supportive and inclusive society for all.\u201d<\/p>\n

As part of the Global Alliance\u2019s commitment to achieving positive change for individuals and families affected by DM, its members will direct their energies toward two primary areas of focus in 2023:<\/p>\n

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  1. Awareness and education among clinical care teams,<\/li>\n
  2. Clinical trial readiness for participants.<\/li>\n<\/ol>\n

    The first area of focus is raising awareness and education among clinical care teams. This will involve working with medical professionals to increase their understanding of DM,\u00a0including its symptoms, diagnosis, and available treatments<\/a>. By doing so, the Global Alliance hopes to improve the quality of care that individuals with DM receive,\u00a0reduce the time to diagnosis<\/a>, and ultimately improve their quality of life.<\/p>\n

    \u201cThe importance of clinicians\u2019 awareness and knowledge of myotonic dystrophy cannot be overstated. Despite its prevalence, myotonic dystrophy is often overlooked due to the variability of the condition and the symptoms overlapping with other diseases. Although it is now confirmable with a simple genetic test, a diagnosis can still be difficult even when DM is suspected,\u201d said Dr. Homira Osman of\u00a0Muscular Dystrophy Canada<\/a>. \u201cIt is common for older family members to only be diagnosed after the birth of a congenitally affected child, which means misdiagnoses can persist for decades and delayed diagnosis is very common. Physicians may see only one or two patients with DM in their entire practice and therefore may be unfamiliar with the varied and complex symptoms that can appear in nearly every system of the body, from locking muscles (myotonia), to heart, breathing, digestive, hormonal, early onset cataracts, and cognitive difficulties, just to name a few.\u201d<\/p>\n

    The second area of focus will be clinical trial readiness for participants. With no existing cure for DM, clinical trials are crucial to the development of new treatments and therapies. However, recruiting participants for\u00a0clinical trials<\/a>\u00a0can be challenging, and the Global Alliance will work to improve access to information about trials and support for those who wish to participate. The Global Alliance will also work to ensure that clinical trials are designed to be inclusive, accessible, and accommodating for all individuals with DM.<\/p>\n

    The Global Alliance for Myotonic Dystrophy Awareness recognizes the importance of clinical trial readiness in the\u00a0quest for therapies and ultimately, a cure for DM<\/a>. \u201cWe must be ready to participate in clinical trials. The goal has to be to provide the greatest potential for finding a cure in the shortest possible time frame for the many people that are suffering, and even at risk of dying,\u201d said Jorg van Gent, Founding Chair of\u00a0MD Nederland<\/a>. \u201cWith over 50 companies and institutions offering significant new investment into research and technologies focused on identifying treatments for myotonic dystrophy, there is hope for improved quality of life, reduced disability, and increased life expectancy for individuals living with this disease within the next few years. These new technologies also hold great promise for individuals with other muscular dystrophies and for conditions with a similar genetic base, such as ALS, Fragile X syndrome and Huntington\u2019s disease.\u201d<\/p>\n

    The Global Alliance encourages and invites all members of the DM community to actively participate in\u00a0Rare Disease Day on February 28th<\/a>,\u00a0DM Families Day on July 29th<\/a>, and\u00a0International Myotonic Dystrophy Awareness Day on September 15th<\/a>. These important days present an opportunity for individuals and organizations to come together and raise awareness of myotonic dystrophy through various activities. These may include sharing personal experiences with friends and family, writing letters to, or meeting with,\u00a0policy makers to advocate for DM research funding and provision of care<\/a>, sharing\u00a0DM clinical care guidelines<\/a>\u00a0with healthcare providers, organizing fundraising events, lighting up monuments and landmarks in green, and more!<\/p>\n

    Through these efforts, the Global Alliance hopes to foster wider recognition and a greater understanding of the disease and to ultimately improve the lives of those affected by it.<\/p>\n

    Together, we are changing the future of myotonic dystrophy.<\/strong><\/p>\n

    ###<\/p>\n

    Established in 2021, the Global Alliance for Myotonic Dystrophy Awareness now includes over 57 international nonprofit organisations, academic and research institutions, biotechnology and pharmaceutical companies, patient advocacy groups, and others working together to raise myotonic dystrophy awareness. Discover DM resources and learn how you can join the movement at:\u00a0https:\/\/www.myotonic.org\/international-dm-day<\/a>.<\/p>\n

    <\/div>\n

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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    <\/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<\/div>\n<\/div>\n","protected":false},"excerpt":{"rendered":"

    Click here to download this press release as a printable PDF. PRESS RELEASE US Contact Name: Tanya Stevenson US Contact Email:\u00a0Tanya.Stevenson@myotonic.org UK Contact Name: Emma-Jayne Ashley UK Contact Email:\u00a0CureDM@outlook.com Netherlands Contact Name: Jorg van Gent Netherlands Contact Email:\u00a0Jorg.vanGent@md.nl Japan Contact Name: Midori Senoo Japan Contact Email:\u00a0Contact@dm-family.net FOR IMMEDIATE RELEASE Global Alliance of 57+ Myotonic Dystrophy […]<\/p>\n","protected":false},"author":9,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[70],"tags":[],"class_list":["post-13228","post","type-post","status-publish","format-standard","hentry","category-news"],"_links":{"self":[{"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/posts\/13228","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/users\/9"}],"replies":[{"embeddable":true,"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/comments?post=13228"}],"version-history":[{"count":0,"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/posts\/13228\/revisions"}],"wp:attachment":[{"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/media?parent=13228"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/categories?post=13228"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/myotonic.org\/de\/wp-json\/wp\/v2\/tags?post=13228"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}