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Myotonic Dystrophy Type 1 (DM1)

Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy in adults and is associated with high morbidity and early mortality.1,2 Symptoms of DM1 are heterogeneous and multi-systemic, affecting skeletal and smooth muscle as well as the eye, heart, endocrine, and central nervous systems.3 Expansions in an unstable CTG repeat region in the DMPK gene lead to a widespread disruption of RNA splicing, known as spliceopathy, which drives the multi-system manifestations of the disease.4Although muscle weakness is often reported as the most prominent and impactful symptom of DM1, many find that CNS-related symptoms are more disabling.5–7

Access the resources below to learn more.
DM1 Disease Background
The modules in this collection provide a comprehensive overview of DM1 presentation, diagnosis, and management.

DM1 modules summary

A one-page summary of the DM1 disease background modules.

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Module 1: What is DM1?

An overview of DM1 and the mechanism of spliceopathy.

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Module 2: How is DM1 diagnosed?

Symptoms and diagnostic pathway for DM1.

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Module 3: What are the clinical manifestations and impact of DM1?

Overview of the multi-system impacts and socio-economic burden of DM1.

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Module 4: What clinical phenotypes are seen in DM1?

Key features of congenital, childhood, adult, and late-onset DM1.

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Module 5: How is DM1 managed and monitored?

Pharmacological and nonpharmacological interventions in DM1, monitoring and the role of the multidisciplinary team.

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Interactive digital resources

Think you know DM1?

An interactive digital resource containing key facts about DM1.

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What is a spliceopathy?

An interactive digital resource explaining what a spliceopathy is, and how it leads to the clinical manifestations of DM1.

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One disease, multiple symptoms

An interactive digital resource exploring the organ systems affected by DM1 and the resulting clinical manifestations.

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References:

1. Liao Q, et al. Neuroepidemiology. 2022;56(3):163–173; 2. Mathieu J, et al. Neurology. 1999;52(8):1658–1662; 3. Ho G, et al. World J Clin Pediatr. 2015;4:66–80; 4. López-Martínez A, et al. Genes (Basel). 2020;11(9):1109; 5. Hagerman KA, et al. Muscle Nerve. 2019;59(4):457–464; 6. Miller JN, et al. Front Neurol. 2021;12:700796; 7. White M. Ther Innov Regul Sci. 2020;54:1010–1017.

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Dyne Therapeutics, Inc. supports accredited continuing education (CE) for healthcare professionals across therapeutic areas of interest. These CE activities are planned, developed and delivered by independent accredited providers in accordance with the standards set forth in the ACCME Standards for Integrity and Independence in Accredited Continuing Education, or the standards of other similar accrediting organizations. Dyne Therapeutics, Inc. does not exercise control over, or provide guidance to, any accredited provider. Dyne Therapeutics, Inc. is sharing CE information on this page as a service to the medical community and with permission from the accredited providers. The information on this page is intended to provide self-service to educational topics of interest, and most activities are free to the learner (some may require a nominal registration fee per the accredited provider’s requirements).

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