Participants who join the extension will continue to take the granules for oral solution each day, with regular clinic visits to check for any side effects and to perform simple functional tests. These tests include the NSAD, a questionnaire that rates everyday activities such as walking and climbing stairs; the 10MWT, which measures how fast a person can walk ten meters; the FVC, a breathing test that estimates lung capacity while seated; the PUL 2.0, an assessment of arm and hand function for those who cannot walk; the 100MTT, a longer walking test; and a blood draw to measure serum CK, an enzyme that rises when muscle tissue is damaged. The schedule is designed to monitor health over an extended period without requiring invasive procedures.

Participants in the study will receive Deflazacort in the form of oral drops, with a dosage of 0.9 mg per kilogram of body weight each day. The study will observe both adult and pediatric patients over a period of time to see how their condition changes with the treatment. The study aims to track changes in clinical outcomes and other health markers, known as biomarkers, to understand the impact of the treatment.

The study will follow participants through several stages, checking their progress at different times to gather data on how Deflazacort affects their condition. This will help researchers determine the effectiveness of the treatment and identify any potential benefits or side effects. The ultimate goal is to find reliable ways to improve the quality of life for those affected by L-CMD.

Participants in the study will be randomly assigned to receive either the BBP-418 treatment or a placebo, which looks like the treatment but does not contain the active substance. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure that the results are not influenced by expectations. The study will last for a period of 36 months, during which participants will undergo regular assessments to monitor their health and the effects of the treatment.

Throughout the study, participants will have various health checks, including physical examinations and laboratory tests, to track any changes in their condition. The main focus will be on changes in muscle strength and function, as well as any side effects that may occur. The study aims to provide valuable information on whether BBP-418 can help improve the symptoms of Limb Girdle Muscular Dystrophy 2I and enhance the quality of life for those affected by this condition.

The purpose of the study is to evaluate the safety of two different doses of ATA-200 when given through an intravenous infusion, which means it is delivered directly into the bloodstream. The study will involve children who can walk and have been diagnosed with LGMDR5. Participants will receive the treatment and be monitored for any side effects or changes in their health. The study aims to find the best dose for future research.

Throughout the study, participants will undergo various assessments to check their muscle function and overall health. These assessments include physical exams, muscle strength tests, and imaging tests like MRI to look at the muscles. The study will also collect information on how the treatment affects the participants’ daily activities and quality of life. The goal is to ensure the treatment is safe and to gather information that could help improve the lives of people with LGMDR5 in the future.

The study is divided into two stages. In the first stage, participants will receive the GNT0006 treatment through an intravenous infusion, which means it will be given directly into a vein. This stage will help determine the best dose of the treatment. In the second stage, the selected dose will be used to further assess how well the treatment works over a period of one year. Some participants may receive a placebo, which is a substance with no active ingredients, to compare the effects of the treatment.

Participants in the study will undergo various assessments to monitor changes in their muscle function and overall health. These assessments may include tests of muscle strength and function, as well as imaging tests like MRI to look at the muscles. The study will also track changes in respiratory function and other health markers. The goal is to see if the GNT0006 treatment can improve the symptoms of FKRP-related limb-girdle muscular dystrophy and enhance the quality of life for those affected by this condition.

The purpose of the study is to understand how EDG-5506 affects markers in the body that indicate muscle damage. Participants in the study will be randomly assigned to receive either the EDG-5506 tablet or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure the results are unbiased.

Throughout the study, participants will undergo various assessments to monitor their health and the effects of the treatment. These assessments will include checking for any side effects, measuring changes in muscle-related markers, and evaluating overall health through tests like blood work and physical exams. The study will last for a period of time, during which participants will be closely monitored to gather important information about the safety and effectiveness of EDG-5506.