An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

To join this study, children must have already completed a previous study (called 111-206) where they received either the study medicine BMN 111 or a placebo.

Parents or guardians need to be willing to give their written permission for their child to take part. If the child is old enough to understand, they will also be asked to agree in writing. It's also important that both the child and their family are able to follow all the study instructions and attend appointments.

However, some reasons might mean a child can't join. This includes if they stopped the previous study early, if they have certain heart problems, or if they're already taking other specific medications that might interfere with the study medicine. Pregnant individuals or those planning to become pregnant cannot participate. The study team will review all health information to make sure it's safe for every child to take part.

If a child joins this study, they will receive BMN 111 as a daily injection under the skin. The dose will depend on their weight and age, as decided during the previous study. The study lasts until each child reaches their estimated adult height, which means it could be several years.

During this time, there will be regular visits to the clinic where doctors will check their health, measure their growth, and possibly take blood or urine samples to see how the medicine is working and to monitor for any side effects. These check-ups are important to make sure the medicine is safe and effective. The exact number and frequency of visits will be explained by the study team, but they will be spread out over the long term.