Duchenne Muscular Dystrophy - Global Clinical Trial Landscape (2024)

Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder causing progressive muscle weakness, affecting about 1 in 5,000 males globally. Higher prevalence is noted in regions like Europe (particularly Sweden and Norway), the US, Canada, and China. Treatment mainly involves glucocorticoids, such as deflazacort, to manage symptoms and slow progression, but recent advances include genetic therapies like exon-skipping drugs (e.g., eteplirsen, golodirsen, viltolarsen) and premature termination codon read-through therapy (ataluren), addressing the dystrophin protein deficiency at the core of DMD.

Since 2019, over 300 clinical trials globally have targeted DMD, with North America and Europe leading. The UK and the US lead in their respective regions, while the Asia-Pacific, spearheaded by Australia and Japan, contributes significantly. Europe shows efficient trial conduct compared to Asia-Pacific and the US.

Ongoing research explores gene and RNA therapies, including gene replacement and exon skipping, alongside cell therapies, membrane stabilization techniques, and interventions for secondary complications. Marketed drugs like Casimersen, Viltolarsen, Deflazacort, and Delandistrogene Moxeparvovec provide tangible options. Overall, this comprehensive approach offers hope for improved outcomes and quality of life for DMD patients, highlighting global commitment to tackling this complex disorder.