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Recent developments and industry interest in gene therapy for duchenne muscular dystrophy
Recent Developments and Industry Interest in Gene Therapy for Duchenne Muscular Dystrophy Introduction Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder that causes progressive muscle degeneration. This condition is caused by mutations in the DMD gene, which encodes a crucial protein for muscle stability. Symptoms usually appear between ages 2 and 5, leading to muscle weakness and, eventually, respiratory or cardiac failure. While antisense oligonucleotides have been approved to …
