Global Prevalence And Clinical Trials Landscape Of Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a rare, debilitating genetic disorder primarily affecting males, caused by a deficiency in the dystrophin protein due to a mutation in the DMD gene. This leads to progressive muscle weakness and degeneration, significantly impacting the quality of life and life expectancy of those affected.

Despite its rarity, the global prevalence of DMD varies due to factors such as genetic diversity, levels of awareness, and the quality of healthcare infrastructure. Current treatment options include glucocorticoids, genetic therapies, and the use of adeno-associated virus vectors to introduce microdystrophin transgenes. Since 2019, around 300 clinical trials have been conducted, with a majority taking place in North America and Europe. As research continues, promising future treatments are being explored, such as cell therapies, membrane stabilization, and the innovative use of RNA-guided CRISPR-Cas9. The development of VBP15, an oral glucocorticoid analog, is also under investigation.

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