ACHONDROPLASIA

This 3-year study is testing whether a combination of two weekly injection drugs can safely help children with achondroplasia grow faster. It involves 22 children aged 2 to 11 and will measure their yearly growth rate and monitor for side effects. The goal is to see if using both…

This study is testing the long-term safety and effectiveness of an experimental drug called BMN 111 in children with achondroplasia, the most common form of dwarfism. The main goal is to see if the drug can safely increase the children's annual growth rate over many years. Resear…

This study continues testing a weekly injection called TransCon CNP in children and teenagers with achondroplasia, a genetic condition that affects bone growth. It's for participants who have already completed an earlier trial with the same medication. The main goals are to check…

This study continues testing a daily injection called BMN 111 in about 70 children with achondroplasia, the most common form of dwarfism. The goal is to see if long-term treatment is safe and helps children grow taller until they reach near-adult height. Researchers will also che…

This study follows children with achondroplasia, a genetic condition that causes short stature, to see how safe and effective a daily injection called BMN 111 is over a longer period. It includes about 30 children who have already been taking the drug for two years in a previous …

This study tests the safety of daily vosoritide injections in infants with achondroplasia (the most common form of dwarfism) who are at risk of needing spinal decompression surgery. Researchers want to see if the treatment is safe for very young children and whether it affects sk…

This study is testing the long-term safety and effectiveness of a daily oral medication called infigratinib in children with achondroplasia, the most common form of dwarfism. It aims to see if the drug can help children grow taller at a rate closer to average, improve their body …