What is exon deletion, and why does it matter in Duchenne muscular dystrophy? An exon deletion occurs when one or more sections of a gene are missing, potentially affecting how the body produces an essential protein. In DMD, these deletions occur in the DMD gene, which contains the instructions...

Understanding the phases of clinical trials can help families make sense of how a potential medicine moves from laboratory research toward possible approval and wider patient access. When families hear that a treatment is in Phase 1, Phase 2, or Phase 3, the terminology can be confusing. Each stage...