The study focuses on Duchenne Muscular Dystrophy, a rare genetic condition that causes progressive muscle weakness. Participants receive an investigational medicine called ENTR-601-45 or ENTR-601-44, which are given by intravenous infusion. These drugs belong to a special class designed to help the body skip over faulty sections of the gene (a process known as exon skipping) and use a delivery system referred to as an endosomal escape vehicle phosphorodiamidate morpholino oligomer platform to reach muscle cells.
The main goal of the trial is to evaluate the long‑term safety and tolerability of the study drug in people with the condition. After an initial screening, participants receive the medication at regular intervals for an extended period, during which doctors monitor vital signs, blood tests, heart recordings, and physical examinations. Simple walking and climbing tests are performed at the start and at later visits to see how the treatment may affect mobility.
Throughout the study, blood samples are taken to measure how much of the medicine remains in the body and to check for any immune response. All observations are recorded to help determine whether the therapy is safe for continued use over time.



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