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.

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.

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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