Exon skipping is a promising therapeutic strategy for Duchenne muscular dystrophy (DMD), a severe genetic disorder. By using antisense oligonucleotides (AOs), this method aims to restore a partially functional dystrophin protein. The study conducted a systematic review and meta-analysis of five randomized controlled trials (RCTs) involving 322 participants, focusing on two drugs: eteplirsen and drisapersen. The analysis evaluated outcomes such as the 6-minute walk test (6MWT), North Star Ambulatory Assessment …

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    1. "Open-label study": Both the participants and the researchers know which treatment is being administered. This design is typically used when it’s not practical or ethical to hide the treatment from the participants, such as in surgical procedures or lifestyle interventions. 2. "Blinded study (single-blind)": In a single-blind study, only the participants are unaware of whether they are receiving the experimental treatment or a placebo (or the standard …

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    Personal take on this article: Recent developments in Becker muscular dystrophy (BMD) research have focused on improving trial readiness and exploring new treatment strategies. Although BMD has historically been overshadowed by Duchenne muscular dystrophy (DMD), recent advances in diagnostics and natural history studies have highlighted the need for more targeted clinical trials. Improved genetic sequencing has led to more frequent diagnoses of BMD, even in diverse populations, and …

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