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 study includes people with Duchenne muscular dystrophy whose gene changes may allow a treatment approach called exon 44 skipping, which means the medicine is designed to help the body work around a missing or faulty part of the gene. The study is planned in two parts. In the first part, treatment is assigned by chance and neither the study team nor the participant knows which treatment is given. Later, there is an open-label extension, which means the treatment is given openly after the blinded part ends. During the study, the medicine is given at regular visits and health checks are done over time.

Participants who join the trial receive the assigned infusion and then return for regular check‑ups over the next 18 months. During these visits, simple tests are performed to measure how quickly a person can stand up, walk a short distance, climb stairs, and breathe fully. A small wearable device may be used to record walking speed in everyday life. Blood and muscle samples may be taken to look for the presence of a protein called microdystrophin, which the therapy is meant to produce. Safety is also monitored by recording any side effects, checking heart rhythm with an ECG, and listening to the lungs with an echo.

The treatment involves intravenous infusion, meaning the medication is delivered directly into a vein through a tube. During this study, participants receive these infusions on a weekly basis. The process is designed to monitor how the body reacts to the medication over an extended period of time to ensure it can be used safely over the long term.

The study will involve boys between 7 and 10 years old who have Duchenne muscular dystrophy confirmed by genetic testing. Participants will need to be taking a stable dose of steroid medicine for at least 3 months before joining the study, or have stopped taking steroids at least 3 months before joining. Those taking other medicines for heart support or supplements that might affect muscles should be on stable doses for at least 1 month before starting. Participants should also be on a stable physical therapy routine for at least 2 months if they are doing such exercises. The study will last for 12 weeks during which participants will take the study medicine or placebo daily.

During the study, doctors will monitor safety by checking for any unwanted effects and by performing physical examinations, blood tests, heart monitoring with ECG, and other assessments. The main measurement of how well the treatment works will be changes in muscle strength tested with special equipment called dynamometry. Other measurements will include changes in muscle tissue examined through magnetic resonance imaging scans of the thigh muscle, a small sample of muscle tissue taken from the upper arm, and various tests of physical function including a test called NSAA that measures ability to perform different movements. Additional measurements will look at markers of muscle damage in the blood, lung function measured by spirometry, and detailed examination of muscle tissue samples to understand changes in muscle fibers and inflammation.

The study will test BMN 351, which is a special type of molecule called a phosphorothioate oligonucleotide that targets a specific part of genetic material called exon 51. The medication is given as a concentrated solution that is diluted and administered directly into the bloodstream through an intravenous line. During the study, participants will receive multiple doses of the medication, and researchers will monitor how the body processes the drug.

Throughout the study, researchers will collect information about any side effects that may occur and perform various medical tests to ensure participant safety. They will also measure how the medication moves through the body and how it affects muscle tissue. This is an open-label study, which means all participants will receive the active medication, and the dose will be gradually increased over time to find the most appropriate amount.

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 investigate the safety and tolerability of a single intravenous dose of SGT-003. Participants will receive the treatment through an infusion into a vein. The study will monitor participants over time to see how their bodies respond to the treatment and to check for any side effects. The study will also look at how the treatment affects the levels and distribution of the microdystrophin protein in the body.

Participants in the study will be ambulant males with DMD, meaning they are able to walk without assistance. The study will track various health indicators, such as muscle function and breathing capacity, to assess any changes from the start of the study. The trial will also examine how the body processes the gene therapy and any immune responses that may occur. The study is expected to continue for several years to gather comprehensive data on the treatment’s effects.

The purpose of the study is to evaluate how safe and effective this new treatment is for patients with DMD. Participants in the study will receive the DT-DEC01 treatment through an injection into the bone. The study will monitor participants over time to observe any side effects and to assess changes in muscle strength and function. This will include tests like the Six-Minute Walk Test, which measures how far a person can walk in six minutes, and other assessments tailored to whether the patient can walk or not.

The study is divided into two phases. In the first phase, the focus is on observing any adverse effects and initial signs of improvement in muscle function. The second phase continues to monitor safety and looks more closely at how well the treatment works over a longer period. Throughout the study, various tests will be conducted to measure muscle strength and quality of life, providing a comprehensive view of the treatment’s impact on patients with Duchenne Muscular Dystrophy.

Participants in the study will be randomly assigned to receive either Metoprolol or a placebo, in addition to their standard heart treatment. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving Metoprolol and who is receiving the placebo. This helps ensure that the results are not biased. The main goal is to determine if Metoprolol can slow the decline in heart function, measured by a test called echocardiography, which uses sound waves to create images of the heart. The study will also look at how long it takes for participants to develop cardiomyopathy or heart failure, which is when the heart can’t pump enough blood to meet the body’s needs.

The trial will involve regular check-ups and heart function tests over a period of time to monitor the effects of the treatment. The study is expected to continue until 2026, providing valuable information on whether Metoprolol can be an effective addition to the treatment plan for young patients with DMD to help protect their heart health.

The purpose of the study is to evaluate the effectiveness and safety of these treatments compared to a placebo. Participants will receive either the active treatment or a placebo without knowing which one they are receiving. The study will monitor changes in the ability to walk, endurance, and overall muscle function over time. The study will also include an open-label extension, where all participants may receive the active treatment after the initial phase. The study will last for a period of time, during which participants will have regular check-ups and assessments to track their progress.

Throughout the study, participants will undergo various tests to measure muscle function, including a walking test known as the 6-Minute Walk Test (6MWT). This test helps assess how far a person can walk in six minutes, which is an indicator of their muscle strength and endurance. The study aims to provide valuable information on how these treatments can potentially benefit individuals with Duchenne Muscular Dystrophy and improve their quality of life.

The purpose of the study is to evaluate how well ATL1102 works in improving upper limb muscle function in participants who are unable to walk due to DMD. The study will compare the effects of the medication to a placebo. Participants will receive the treatment once a week, and the study will monitor their muscle function over time. The study will also assess the safety and tolerability of the medication to ensure it is safe for use.

The study is divided into two phases: a blinded phase, where neither the participants nor the researchers know who is receiving the actual medication or the placebo, and an open-label extension phase, where all participants will receive ATL1102. The study will last for several weeks, and participants will be regularly assessed to track changes in their muscle function and overall health. The goal is to determine if ATL1102 can help improve the quality of life for those living with Duchenne Muscular Dystrophy.

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, all participants will receive the same investigational drug, GIVINOSTAT. The study will monitor the type, frequency, and severity of any side effects that may occur. For patients who can walk, changes in physical function and muscle strength will be assessed. For those who cannot walk, changes in physical function, daily living activities, and upper limb strength will be evaluated. Additionally, the study will track changes in respiratory function and quality of life for all participants.

The study will continue over several years, with assessments taking place at specific intervals, such as after 48 weeks and then annually. The goal is to gather comprehensive data on how GIVINOSTAT affects patients with Duchenne Muscular Dystrophy over an extended period. This information will help determine the long-term benefits and risks of using GIVINOSTAT as a treatment option for this condition.

The purpose of this study is to evaluate how effective Satralizumab is in improving bone health, specifically looking at bone mineral density, which is a measure of bone strength. The study will also assess the safety of the medication and how it interacts with the body. Participants in the study will receive either Satralizumab or a placebo, and their progress will be monitored over a period of time to see how the treatment affects their condition.

Throughout the study, participants will undergo various assessments to track changes in their bone health and overall well-being. These assessments will include regular check-ups and tests to measure bone density and other health indicators. The study aims to provide valuable information on the potential benefits and safety of Satralizumab for young patients with Duchenne Muscular Dystrophy.

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 study will involve five participants who are unable to walk due to DMD. Each participant will receive an injection of the genetically corrected MABs into a muscle in the foot. The researchers will monitor the participants for any adverse effects and will also check if the treatment successfully increases the levels of dystrophin in the muscle. The study will last for one year, during which the safety and effectiveness of the treatment will be closely observed.

Participants will be monitored for any local or systemic adverse events following the injection. The primary goal is to ensure the treatment is safe and to determine if there is an increase in dystrophin levels in the muscle, which will be assessed through muscle biopsies. The study is designed to provide valuable information on the potential of this innovative treatment for improving muscle function in individuals with Duchenne Muscular Dystrophy.

The purpose of this study is to gather information on the safety and effectiveness of long-term treatment with vamorolone, particularly in relation to bone health, such as the risk of vertebral fractures. Participants in this study are boys who have previously been involved in other studies with vamorolone and are continuing their treatment. The study will monitor various health aspects over time, including the occurrence of fractures, changes in body weight and height, and other health markers.

Throughout the study, participants will undergo regular assessments to track their progress and any potential side effects. These assessments will help researchers understand how vamorolone affects the body over an extended period. The study aims to provide valuable insights into the long-term use of vamorolone for managing symptoms of Duchenne Muscular Dystrophy.

The purpose of the study is to understand how Givinostat is processed in the body and to evaluate its safety when used over a long period. The study is divided into two phases: the Core Phase and the Extension Phase. In the Core Phase, participants will receive Givinostat to see how their bodies absorb and process the medication. In the Extension Phase, the focus will be on monitoring the safety and any side effects of the medication over a longer duration.

Participants in the study will be young boys aged between 2 and less than 6 years old who have been diagnosed with DMD. The study will involve regular check-ups and assessments to monitor the effects of Givinostat on the participants’ health and well-being. The trial aims to provide valuable information on the potential benefits and risks of using Givinostat in managing Duchenne muscular dystrophy in young children.

The purpose of the study is to evaluate the safety and effectiveness of different doses of eteplirsen. The study begins with an open-label phase, where all participants receive increasing doses of eteplirsen to assess how well they tolerate the treatment. Following this, there is a double-blind phase, where participants are randomly assigned to receive either a high dose or a lower dose of eteplirsen, or a placebo, without knowing which one they are receiving. This phase aims to compare the effects of the different doses on the motor function of patients who can walk and have the specific genetic mutations suitable for exon 51 skipping.

Throughout the study, participants will receive weekly intravenous infusions of eteplirsen. The study will monitor various health indicators, including motor function tests and safety assessments, over a period of time to determine the impact of the treatment. The study is expected to continue until 2026, providing valuable information on the potential benefits and risks of eteplirsen for treating Duchenne Muscular Dystrophy.

The purpose of this study is to evaluate the safety and tolerability of Viltolarsen when given intravenously, meaning directly into a vein, at weekly doses. Participants in this study are boys who have previously completed another study involving Viltolarsen. The study will monitor various health indicators, such as vital signs and physical examinations, to ensure the treatment is safe and well-tolerated.

Throughout the study, participants will undergo several assessments to track their progress. These assessments include tests like the Time to Stand Test, which measures how quickly a participant can stand up, and the Six-minute Walk Test, which evaluates how far a participant can walk in six minutes. These tests help researchers understand the effects of Viltolarsen on muscle strength and function in boys with Duchenne Muscular Dystrophy.

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 main purpose of this research is to test how safe DYNE-251 is and how well patients tolerate it when given multiple doses. The study will also measure the levels of a protein called dystrophin in muscle tissue after treatment. This protein is important because people with Duchenne Muscular Dystrophy don’t make enough of it.

During the study, participants will receive regular infusions of either DYNE-251 or placebo. The treatment schedule will vary, with some participants receiving treatment every four weeks and others every eight weeks. The study includes various health checks and muscle tissue examinations to monitor how well the treatment is working. Doctors will track any changes in muscle function and strength throughout the study period.

The purpose of the study is to evaluate the safety and effectiveness of Fordadistrogene Movaparvovec in treating DMD. Participants in the study will receive the treatment through an intravenous infusion, which means it will be delivered directly into the bloodstream. The study will last for a period of time during which participants will be monitored for changes in their muscle function and overall health. The main goal is to see if the treatment can improve the ability to perform daily activities and increase muscle strength.

In addition to the main treatment, the study will also involve the use of Eculizumab, another medication that will be administered as a concentrate for infusion. This medication is being used to help understand its effects in combination with the main treatment. Throughout the study, participants will undergo various assessments to track their progress, including tests to measure muscle strength and function. The study aims to provide valuable information on the potential benefits of gene therapy for individuals with DMD.

The trial is designed for children and adolescent males with genetically confirmed DMD who use wheelchairs. Participants will receive either givinostat or placebo for 18 months. During this time, their upper limb function will be assessed using a standardized test called the Performance of the Upper Limb test. Additionally, breathing capacity will be monitored through measurements of Peak Expiratory Flow (the maximum speed of breath when exhaling) and Forced Vital Capacity (the total amount of air that can be forcibly exhaled).

Throughout the study, researchers will track how the medication affects muscle function in the arms and hands, as well as respiratory function. They will also monitor for any side effects that might occur during treatment, including changes in vital signs, laboratory test results, and heart function.

The purpose of the study is to gather information on the safety and tolerability of SQY51 in both children and adults who have a genetically confirmed diagnosis of DMD. The study is divided into two phases: a 13-week Phase 1, where multiple doses of the treatment will be given to understand how the body processes the drug, and a 32-week Phase 2a, which will continue to monitor the effects of the treatment over a longer period. Participants will receive the treatment through intravenous use, meaning it will be delivered directly into the bloodstream.

Throughout the study, various health assessments will be conducted to monitor the participants’ response to the treatment. These assessments will include checking for any adverse events, conducting physical examinations, and performing tests such as electrocardiograms (ECGs) to evaluate heart function. Additionally, the study will measure motor function, respiratory function, and muscle strength to understand the impact of SQY51 on the symptoms of DMD. The study will also analyze biomarkers in the blood and muscle tissue to gain further insights into the treatment’s effects. The trial is expected to conclude in 2025.

The study is divided into three parts. The first part aims to find the right dose of GNT0004 that is both safe and effective. Once the appropriate dose is determined, the second part of the study will compare the effects of GNT0004 with a placebo to evaluate its safety and effectiveness over a year. The final part of the study will continue to monitor the long-term safety and effectiveness of the treatment.

Participants in the study will receive the treatment through an infusion, which is a method of delivering medication directly into the bloodstream. The study will help researchers understand how well GNT0004 works in treating Duchenne Muscular Dystrophy and its potential benefits for improving muscle function in affected boys. The trial will also monitor any side effects or adverse reactions to ensure the treatment is safe for long-term use.