The study focuses on Duchenne Muscular Dystrophy, a rare genetic condition that leads to progressive muscle weakness and loss of function. Participants who can benefit from a technique called exon skipping—a method that allows cells to produce a shorter but still useful form of the missing protein—will receive one of two investigational medicines, ENTR-601-45 or ENTR-601-44. Both drugs are delivered by intravenous infusion, meaning they are given slowly through a needle placed in a vein, and they belong to a phosphorodiamidate morpholino oligomer platform designed to help the therapeutic molecules reach muscle cells.
The purpose of the trial is to evaluate the long‑term safety and tolerability of the study drug in this patient population. After an initial screening, participants will receive the assigned medication at regular intervals over an extended period while undergoing routine health checks, including measurements of vital signs, blood laboratory tests, and a heart test known as an electrocardiogram. Physical examinations will assess walking ability, standing up from the floor, climbing stairs, and upper‑limb function, and blood samples will be taken to monitor drug levels and any immune response. The study is open‑label, so all participants know they are receiving the investigational therapy, and it continues beyond the earlier phase of the research.
Belgium 
Italy 
Spain 
The Netherlands 