Exon 51 specific phosphorothioate oligonucleotide clinical trials

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

This study is for men with Duchenne muscular dystrophy, aged 18 and over. It's looking into the long-term safety and how well a new medicine, BMN 351, works. This medicine is given weekly by drip and helps with a specific type of Duchenne.