The study has two parts. In the first part, different doses of BMN 333 are compared with vosoritide. In the second part, one selected dose of BMN 333 is compared with vosoritide. The study lasts about one year for each child after treatment starts. During the study, treatment is given as a shot under the skin, and regular checkups are done to follow the child’s growth and overall health.

The study looks at growth over time, including annual growth velocity, which means how much a child grows in one year. It also follows body measurements and checks for side effects, including changes in heart tracing tests, blood tests, and X-rays. These checks are used to help understand both the benefits and the risks of the medicines being studied.

The purpose of this study is to assess the long-term safety and effectiveness of BMN 111 in promoting growth in children with achondroplasia. Participants in the study will receive regular injections of the medication and will be monitored over an extended period. The study will track growth rates and any potential side effects to ensure the treatment is both safe and beneficial for the children involved.

Throughout the study, various health checks will be conducted, including physical exams and imaging tests like X-rays, to observe changes in bone growth and overall health. The study aims to provide valuable information on how BMN 111 can help children with achondroplasia achieve better growth outcomes and improve their quality of life.

The purpose of the study is to find out if infigratinib is safe and works well in babies and young children with achondroplasia who are younger than three years old. The study is divided into several parts. The first part will test different single doses of the medication to find the right dose to use in later parts. The next part will confirm that the chosen doses are safe and will check how the medication moves through the body. The main part of the study will compare infigratinib to placebo over a period of time to see how well it works and whether it is safe. After completing the main part, children can continue in an extension part of the study until they reach three years of age.

During the study, children will be checked regularly for any side effects and changes in their health. Doctors will measure body length and compare it to growth charts made specifically for children with achondroplasia. They will also measure the ratio between the upper and lower parts of the body, head size compared to body length, and will check bone development using x-rays. Some children will have an MRI scan to look at skull and brain shape. The study team will track when the soft spots on the head close, monitor sleep problems, check if any surgeries are needed, and assess the child’s development of movement, language, thinking, and social skills. Parents or guardians will also answer questions about the child’s quality of life.

The purpose of this study is to evaluate the long-term safety and effectiveness of Infigratinib in children with Achondroplasia. Participants in the study will receive daily doses of the medication over an extended period. The study will monitor changes in the children’s growth, specifically looking at their height over time, as well as any potential side effects that may occur. The study aims to understand how Infigratinib can help improve growth and overall health in children with this condition.

Throughout the study, various aspects of the children’s health will be assessed, including their body proportions, weight, and any changes in their physical abilities. The study will also look at the impact of the medication on the children’s quality of life, including their ability to perform daily activities and any changes in their cognitive functions. The study is designed to provide valuable information on the potential benefits and risks of using Infigratinib for treating Achondroplasia in children.

Participants in the study will receive weekly injections of either TransCon CNP or a placebo for a period of 52 weeks. 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 unbiased. The main goal is to see if the treatment can improve growth over the course of the study.

Throughout the study, participants will be monitored for any changes in their height and overall health. The study aims to provide valuable information on the safety and effectiveness of TransCon CNP in treating achondroplasia, potentially offering a new option for managing this condition in adolescents.

Participants in the study will receive weekly injections of either TransCon CNP or a placebo for a period of 52 weeks. 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 during this time. After the initial 52 weeks, there will be an open-label extension period where all participants may receive the actual treatment. The study aims to observe changes in growth over the course of the trial.

The trial is specifically for children aged 2 to 11 years who have been diagnosed with Achondroplasia. The main goal is to see if the treatment can increase the rate of growth in these children. The study will also look at changes in height compared to the baseline measurements taken at the start of the trial. This research is important for understanding how TransCon CNP might help children with this growth disorder in the future.

The purpose of the study is to evaluate how effective and safe Infigratinib is for children aged 3 to under 18 years who have Achondroplasia and still have the potential to grow. The study will compare the effects of Infigratinib to a placebo, which is a substance with no active medication. Participants will be randomly assigned to receive either Infigratinib or the placebo, and neither the participants nor the researchers will know who is receiving which treatment. This is known as a double-blind study. The treatment period will last up to 52 weeks, during which various measurements related to growth and development will be taken to assess the medication’s impact.

Throughout the study, researchers will monitor changes in growth rates, body proportions, and other health indicators to determine the effectiveness of Infigratinib. The study will also track any side effects or adverse events to ensure the safety of the participants. By the end of the study, the goal is to gather enough information to understand how Infigratinib can help children with Achondroplasia grow and develop more effectively.

The purpose of the study is to assess how safe and well-tolerated TransCon CNP is, as well as its effect on growth in young children with achondroplasia. Participants in the study will receive weekly injections of either TransCon CNP or a placebo for a period of 52 weeks. After this period, there will be an open-label extension, where all participants may receive the actual medication. The study aims to observe any side effects and measure changes in growth over time.

Throughout the study, various assessments will be conducted to monitor the health and development of the participants. These assessments will include measuring growth rates and evaluating any potential side effects. The study will also use imaging techniques like MRI to examine changes in bone structure and other physical characteristics associated with achondroplasia. The goal is to gather comprehensive data on how TransCon CNP affects growth and overall health in children with this condition.

Participants in the study will receive the treatments once a week over a period of 156 weeks, which is about three years. The study will monitor the children’s growth and overall health during this time. The researchers will compare the effects of using both treatments together to using Navepegritide alone. This will help determine if the combination of treatments is more effective in promoting growth.

Throughout the study, the safety of the treatments will be closely observed. This includes checking for any side effects and monitoring the children’s health through regular medical assessments. The study aims to provide valuable information on the potential benefits and safety of these treatments for children with Achondroplasia.

Participants in the study will receive daily injections of BMN 111 and will be monitored for any side effects or changes in their health. The study will also look at how the treatment affects growth, including height and body proportions, to see if it helps improve these aspects in children with achondroplasia. The study is designed to gather information over a long period to ensure the treatment is safe and effective for long-term use.

Throughout the study, various health checks will be conducted, such as physical exams and imaging tests, to ensure the participants’ well-being. The study aims to provide valuable insights into the potential benefits and risks of using BMN 111 for treating achondroplasia in children, contributing to better management of the condition in the future.

The purpose of the study is to understand how safe and tolerable SAR442501 is for children with Achondroplasia. Participants will receive the treatment through subcutaneous injections, which means the medicine is injected just under the skin. The study will take place over a period of time, and participants will be monitored for any side effects or changes in their condition. The study will also look at how the treatment affects growth and development in children with Achondroplasia.

Throughout the study, researchers will collect information on various aspects of the participants’ health, including growth rates, body proportions, and overall quality of life. This information will help determine how the treatment works in the body and its potential benefits for children with Achondroplasia. The study aims to provide valuable insights into the treatment’s effectiveness and safety, contributing to better care for those with this condition.

Participants in the study will receive TransCon CNP through a subcutaneous injection, which means it is injected under the skin. This will be done once a week. The study is designed to observe how well the treatment is tolerated by the participants and to monitor any side effects that may occur. Additionally, the study aims to assess the impact of the treatment on the growth of children and adolescents with achondroplasia.

The trial will continue for an extended period to gather comprehensive data on the long-term effects of TransCon CNP. Throughout the study, various health assessments will be conducted, including physical examinations and other safety checks, to ensure the well-being of the participants. The ultimate goal is to determine if TransCon CNP can be a safe and effective option for improving growth in individuals with achondroplasia.