The purpose of the study is to assess the long‑term safety, tolerability, and effectiveness of the medicines in participants with the condition. Participants receive the study drug at regular intervals over an extended period and attend scheduled visits where vital signs, blood tests, heart rhythm checks (ECG), and physical examinations are performed. Simple walking and climbing tests are used to see how muscle function changes over time. The study also looks at the drug’s pharmacokinetics – how the medication is absorbed, distributed, and cleared from the body – and monitors for any immune response such as anti‑drug antibody formation.

The purpose of this research is to determine if ENTR-601-45 is safe and well-tolerated in people with Duchenne Muscular Dystrophy who have a specific genetic change that can be treated by skipping a part of the gene called exon 45. The study is divided into two parts, with the first part testing different dose levels of the medication to understand how it works in the body and how safe it is.

During the study, participants will receive regular infusions of either ENTR-601-45 or placebo. The research team will monitor the participants’ health through various tests, including muscle tissue samples to see how the treatment affects the muscles. Neither the participants nor the study doctors will know who is receiving the actual medication or the placebo during the study period.

The study is divided into two parts and uses both the investigational medication and sodium chloride solution as placebo. During the study, participants receive either ENTR-601-44 or placebo through an infusion into their veins. The researchers will monitor the participants’ health, including physical examinations, laboratory tests, and measurements of muscle strength and function.

Throughout the study, doctors will collect various types of information about how the medication affects the body, including its presence in blood, muscle tissue, and urine. They will also measure changes in a protein called dystrophin in muscle tissue and monitor the participants’ ability to perform various physical activities. The study includes regular medical check-ups to ensure participant safety.

The purpose of the study is to evaluate the safety of delandistrogene moxeparvovec in young children under the age of four who have been diagnosed with Duchenne Muscular Dystrophy. Participants will receive the treatment through an intravenous method, which means it will be administered directly into a vein. The study will monitor the participants for any side effects or changes in their health, including any serious adverse events or significant changes in vital signs and laboratory assessments.

The study will also measure the amount of dystrophin protein produced in the body after receiving the treatment, specifically looking at changes from the start of the study to 12 weeks later. This will help researchers understand how well the treatment works in increasing dystrophin levels, which is crucial for improving muscle function in individuals with Duchenne Muscular Dystrophy.

During the study, participants will receive the treatments through an intravenous infusion, which means the medication is delivered directly into the bloodstream. The study will monitor changes in the amount of dystrophin protein in muscle tissue, as this protein is crucial for muscle function. The study will also assess the presence of the gene therapy in muscle tissue and the body’s response to the treatment over time. Participants will be observed for any side effects or adverse reactions to ensure the treatment is safe and well-tolerated.

The study will take place over several weeks, with regular check-ups and assessments to track the progress and effects of the treatment. The goal is to determine if the combination of Delandistrogene moxeparvovec and Imlifidase can improve muscle function and slow down the progression of Duchenne muscular dystrophy in those with specific antibodies. This research could provide valuable insights into new treatment options for individuals living with this challenging condition.

The purpose of the study is to assess the safety and effectiveness of Delandistrogene Moxeparvovec in both non-ambulatory (those who cannot walk) and ambulatory (those who can walk) participants with Duchenne Muscular Dystrophy. Participants will receive the treatment through an intravenous infusion, which means the solution is delivered directly into the bloodstream. The study will monitor changes in muscle function and other health indicators over a period of time, with key assessments occurring at 72 weeks after the start of the trial.

Throughout the study, researchers will track various health outcomes, including muscle strength and respiratory function, to determine the impact of the treatment. The trial will also measure the amount of dystrophin, a protein that is typically lacking in individuals with Duchenne Muscular Dystrophy, produced by the body after receiving the gene therapy. Participants will be closely monitored for any side effects or adverse reactions to ensure their safety during the trial.

The purpose of this study is to assess the long-term safety of Delandistrogene Moxeparvovec in participants who have previously received this treatment in an earlier clinical study. Participants will be monitored over a period of time to observe any potential side effects or adverse events that may occur after receiving the treatment. The study will also track changes in participants’ physical abilities, such as their ability to walk, run, and perform upper limb tasks, as well as their lung function and heart health.

Throughout the study, participants will undergo various assessments, including the North Star Ambulatory Assessment and Magnetic Resonance Imaging (MRI), to evaluate changes in their condition from the time before they received the treatment to five years after. This long-term follow-up is crucial to understanding the overall impact of Delandistrogene Moxeparvovec on individuals with Duchenne Muscular Dystrophy.

The study is divided into two parts. In the first part, participants will receive different doses of Vesleteplirsen to find the highest dose that can be tolerated safely. This part will help researchers understand how the body handles the medication and its safety profile. In the second part, participants will receive the dose determined from the first part, and researchers will measure the amount of dystrophin in the muscle tissue to see how well the treatment is working. The medication is given every four weeks, and the study will monitor participants over a period of time to gather comprehensive data.

Throughout the study, researchers will also keep track of any side effects or adverse events that participants may experience. This information is crucial for understanding the safety and effectiveness of Vesleteplirsen in treating Duchenne Muscular Dystrophy. The study is designed to provide valuable insights into how this treatment can potentially improve muscle function and quality of life for those affected by this condition.