Dyne Medical Central

Welcome to Dyne Medical Central

At Dyne Therapeutics, we are focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. Dyne Medical Central is an educational hub designed to help healthcare professionals deepen their understanding of Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and facioscapulohumeral muscular dystrophy (FSHD).

Explore a variety of accessible, downloadable resources to expand your clinical knowledge!

DMD

Duchenne muscular dystrophy (DMD) is a rare, X-linked genetic disorder caused by mutations in the DMD gene, resulting in little or no dystrophin protein. DMD typically presents in early childhood and leads to progressive muscle weakness, loss of mobility and motor function, and serious long-term health issues like cardiac and respiratory complications.1

Explore comprehensive educational resources on DMD, including detailed explanations of pathophysiology, early clinical signs, diagnostic approaches, and best practices for clinical assessment.

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DM1

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy in adults. DM1 is caused by an abnormal expansion of CTG repeats in the DMPK gene, causing RNA mis-splicing (also called a spliceopathy). This multi-system genetic disorder affects skeletal and smooth muscle, the central nervous system, eyes, heart, and several other organ systems. People living with DM1 may experience muscle weakness, myotonia (delayed muscle relaxation), fatigue, daytime sleepiness, cognitive impairment, and a wide range of systemic complications.2,3

Discover educational modules and expert videos on DM1 disease mechanism, spectrum of clinical presentation, patient burden, and multi-disciplinary management.

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FSHD

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic, progressive muscle neuromuscular disorder characterised by gradual and often asymmetric muscle weakness. Weakness usually begins in the facial, shoulder, and upper arm muscles, but can extend to other muscle groups as the disease progresses. Clinical presentation can vary widely between individuals. FSHD is driven by aberrant expression of the DUX4 gene, which leads to muscle inflammation, cell damage, and gradual muscle atrophy.4

Explore educational modules on FSHD disease mechanism and clinical evaluation.

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Recent Content

  • 5-Times Sit-To-Stand (5xSTS): A Comprehensive, Multi-System Measure Of Function In DM1

    An infographic highlighting the 5-times-sit-to-stand test as a potential comprehensive, multi-system measure of functional improvement in DM1.

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  • CNS Manifestations In DM1

    Explore this resource to understand how DM1 affects the brain and the impact of CNS-related symptoms on affected individuals.

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  • Functional improvement: Moving beyond dystrophin in DMD

    Explore this short presentation that aims to define how we approach patient outcomes in DMD.

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  • Living with Duchenne muscular dystrophy

    Watch this video following the journey of Conner and his mother, Jessica, offering a personal perspective on living with DMD.

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Upcoming Events

Stay connected with Dyne Therapeutics at upcoming scientific congresses and educational events focused on DMD, DM1 and FSHD.

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References:

1. Birnkrant DJ, et al. Lancet Neurol. 2018;17(3):251–267; 2. Liao Q, et al. Neuroepidemiology. 2022;56(3):163–173; 3. Ho G, et al. World J Clin Pediatr. 2015;4(4):66–80; 4. Tihaya MS, et al. Nat Rev Neurol. 2023;19(2):91–108.