The purpose of this research is to understand how apitegromab works in young children with SMA and to evaluate its effects on their movement abilities. All children in the study must already be receiving or have received other approved treatments for SMA, such as onasemnogene abeparvovec-xioi, nusinersen, or risdiplam.

The study will last for 48 weeks, during which children will receive regular infusions of the study medication. Throughout the study, doctors will monitor how the medicine moves through the body and its effects on muscle function. This is a double-blind study, which means neither the participants nor the doctors directly involved in their care will know which treatment each child receives.

The study will be conducted in two parts. In the first part, healthy adult male participants will receive a single dose of BIIB115 through an injection into the space around the spinal cord, a method known as intrathecal injection. In the second part, children with SMA will receive multiple doses of BIIB115 using the same method. The study aims to monitor any side effects and how the body processes the medication over time. Participants will be observed for any adverse events, which are any unwanted effects that occur during the study.

The trial will also measure the concentration of BIIB115 in the participants’ blood and spinal fluid to understand how the drug is absorbed and eliminated by the body. This information will help determine the appropriate dosage and frequency of administration for future treatments. The study is expected to continue for several years to gather comprehensive data on the safety and effectiveness of BIIB115 in treating Spinal Muscular Atrophy.

Participants in this study will be monitored over an extended period to observe any serious side effects or specific adverse events that may arise from the treatment. The study aims to understand the long-term effects of OAV101 on patients with SMA, focusing on safety aspects. The trial will track the number and proportion of patients experiencing these events throughout the study duration, which can last up to 15 years.

In addition to safety, the study will also look at developmental milestones, which are key skills or abilities that children typically achieve as they grow. Researchers will assess how many participants reach these milestones and whether they maintain them over time. Changes in motor function will be measured using specific scales, such as the Hammersmith Functional Motor Scale – Expanded and the Revised Upper Limb Module. The study will also monitor vital signs and laboratory values to identify any significant changes that could be related to the treatment.

The purpose of this study is to evaluate the safety and effectiveness of these treatments when used together. Participants in the study will receive either the combination of RO7204239 and Risdiplam or a placebo, which is a substance with no active medication. The study will be conducted in two parts. In the first part, the focus will be on understanding how the body processes these medications and their safety. The second part will assess how well the combination works in improving muscle function in people with Spinal Muscular Atrophy.

Throughout the study, participants will undergo various assessments to monitor their health and the effects of the treatment. These assessments may include regular check-ups, blood tests, and imaging tests like MRI to evaluate muscle changes. The study aims to provide valuable information on the potential benefits of combining RO7204239 and Risdiplam for individuals living with Spinal Muscular Atrophy.

The main purpose is to evaluate how well clenbuterol works and how safe it is when used as a long-term treatment for patients with SBMA. During the study, participants will receive either clenbuterol tablets or placebo tablets for a period of 48 weeks. The medication will be taken by oral route, with a maximum daily dose of 40 micrograms.

Throughout the study, various measurements will be taken to assess how participants respond to the treatment, including their ability to walk and perform daily activities. The study will also monitor breathing function and overall quality of life. Regular check-ups will be conducted to ensure participant safety and to track any changes in their condition.

The medication is given as tablets that participants take by mouth twice daily for 21 days. During the study, some participants will receive NMD670 tablets while others will receive placebo tablets. After a break period, participants who first received the study medication will switch to placebo, and those who started with placebo will receive NMD670. This type of study design helps researchers better understand how well the medication works.

The main focus is on measuring how far participants can walk in 6 minutes before and after taking the medication. The study also looks at other aspects of muscle function and strength, as well as the safety of the medication. Throughout the study, participants’ health will be monitored through various medical tests and examinations to ensure their safety and evaluate how well the treatment is working.

During the study, infants will receive Risdiplam as an oral solution, which can also be administered through a nasogastric tube or a gastrostomy tube if needed. The study will monitor the infants over a period to observe how their bodies handle the medication and to check for any side effects. The study aims to gather important information about the medication’s safety and how it behaves in the body, which is known as pharmacokinetics.

Participants in the study will be closely observed for any changes in their health, including monitoring vital signs like heart rate and blood pressure, as well as conducting physical examinations. The study will also track any adverse events or unusual laboratory results to ensure the safety of the infants involved. This research is crucial for understanding the potential benefits and risks of Risdiplam in treating infants with Spinal Muscular Atrophy.

Participants in the study will receive risdiplam for a period of time, and the effects on their motor skills will be monitored. The study will look at changes in the participants’ ability to move and perform physical activities over the course of the treatment. The treatment is given in a liquid form that can be taken by mouth or through a feeding tube if necessary. The study aims to provide valuable information on how risdiplam can help improve the quality of life for children with SMA.

Throughout the study, researchers will carefully observe the participants to ensure their safety and to gather data on how well the treatment works. The study is designed to last for a specific period, during which the participants’ progress will be regularly assessed. This research is important for understanding how early intervention with risdiplam can benefit children with SMA who have already received gene therapy, potentially leading to better outcomes for those affected by this condition.

The purpose of the study is to assess how well risdiplam works in improving the condition of children with SMA who have not shown further improvement or have worsened after their initial gene therapy. Participants will receive risdiplam for a period of time, and their progress will be monitored to see if there are any changes in their motor skills and overall function. The treatment is administered as a liquid that can be taken by mouth or through a feeding tube if necessary.

Throughout the study, the health and safety of the participants will be closely observed. The study aims to provide valuable information on whether risdiplam can help children with SMA regain or improve their physical abilities after experiencing a decline following gene therapy. This research is important for understanding how to better support children with this condition and improve their quality of life.

The purpose of this study is to evaluate how well patients tolerate apitegromab over an extended period. Participants will receive regular infusions of the medication and will be monitored for any side effects or changes in their condition. The study will track various health measures to understand the impact of the treatment on muscle function and overall health. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of apitegromab.

Throughout the study, participants will have regular check-ups and assessments to ensure their safety and to gather information on how the treatment is working. The study aims to provide valuable insights into the long-term use of apitegromab for managing SMA, helping to improve future treatment options for individuals with this condition.

The purpose of the study is to gather information on the safety and effectiveness of AVXS-101 over an extended period. Participants in this study have previously received AVXS-101 in earlier clinical trials. The study will monitor various aspects of the participants’ health, including their ability to reach developmental milestones, changes in motor skills, and overall physical health. Researchers will also observe any significant changes in areas such as breathing, swallowing, and heart function.

Throughout the study, participants will undergo regular assessments to track their progress and any potential side effects. These assessments will help researchers understand how the treatment impacts the participants’ health over time. The study aims to provide valuable insights into the long-term benefits and risks of using AVXS-101 for treating Spinal Muscular Atrophy.

Participants in the study may already be taking other SMA treatments like nusinersen, risdiplam, or have a history of using onasemnogene abeparvovec-xioi. The study will compare the effects of Taldefgrobep Alfa to a placebo over a period of 48 weeks. During this time, changes in muscle function will be measured to see if the treatment helps improve movement abilities.

The study will also monitor the safety of Taldefgrobep Alfa by checking for any side effects or changes in body composition, such as muscle and bone health. Participants will receive regular assessments to ensure the treatment is well-tolerated. The trial includes an open-label extension, meaning that after the initial study period, all participants may have the opportunity to receive Taldefgrobep Alfa if they choose to continue in the study.

During the study, infants will receive Risdiplam for a period of up to 60 months. The study will monitor the infants’ ability to achieve certain developmental milestones, such as sitting without support, over the course of the treatment. The study will also look at other aspects of the infants’ health, including their ability to swallow and feed orally, as well as their growth and development. The study aims to see if Risdiplam can help improve these areas for infants with SMA.

Throughout the study, various devices will be used to accurately measure and administer the oral solution. These include special dispensers and bottle adapters that ensure the correct amount of medication is given. The study will also track any side effects or adverse events that may occur during the treatment period. The ultimate goal is to determine if Risdiplam can provide a meaningful benefit to infants with SMA, potentially improving their quality of life and developmental outcomes.

The purpose of the study is to evaluate the safety and how well the body tolerates Risdiplam in both adults and children who have SMA. Participants in the study will receive Risdiplam and will be monitored over a period of time to see how their bodies respond to the treatment. The study will also look at how Risdiplam is processed in the body, which is known as pharmacokinetics, and how it affects the body, known as pharmacodynamics.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. This includes looking at any side effects they might experience and measuring levels of certain proteins in the blood that are related to SMA. The study aims to gather important information that could help improve treatment options for people living with SMA in the future.

Participants in the study will receive Risdiplam as an oral solution, which means it is taken by mouth. The study will track changes in the participants’ clinical and functional health from the start of the study to the end of the 24-month period. This includes looking at how well the patients can perform daily activities and any changes in their muscle function. Additionally, the study will monitor certain substances in the blood, known as biomarkers, to see how they change with treatment. These biomarkers include neurofilament light chain and neurofilament heavy chain, which are proteins that can provide information about nerve health.

The study will also assess the safety and tolerability of Risdiplam, meaning it will look at how well patients can handle the medication and any side effects they might experience. The goal is to gather comprehensive information on how Risdiplam affects adults with SMA over the long term, providing valuable insights into its benefits and any potential risks. Participants will be followed closely throughout the study to ensure their safety and to collect detailed data on their health outcomes.