Achondroplasia

This study tests a weekly injection called TransCon CNP in children and teens with achondroplasia, the most common form of dwarfism. The goal is to see if it is safe and helps improve growth over several years. Participants who completed a previous TransCon CNP trial can join thi…

This study tests a daily injection called BMN 111 in about 70 children with achondroplasia, the most common form of dwarfism. The goal is to see if the drug is safe and helps children grow taller over several years. All participants had already received BMN 111 or a placebo in a …

This study tests a drug called vosoritide in infants with achondroplasia (the most common form of dwarfism) who are at high risk of needing a surgery to relieve pressure on the spinal cord at the base of the skull. The main goal is to see if the drug is safe and if it can help en…

This study tests a drug called BMN 111 in children with achondroplasia, the most common form of dwarfism. The goal is to see if it safely helps them grow taller over several years. About 119 children who completed an earlier study will take the drug and be monitored for growth, d…

This study looks at the long-term safety and effects of a daily injection called BMN 111 in children with achondroplasia, a common form of dwarfism. It includes 30 children who have already taken the drug for two years in a previous study. The goal is to see if the drug remains s…

This study tests whether giving two weekly injections together can improve growth in children aged 2 to 11 with achondroplasia, a common cause of dwarfism. The drugs, navepegritide and lonapegsomatropin, are designed to increase height. The trial will measure growth rate and moni…

This study tests a daily oral drug called infigratinib in 300 children with achondroplasia, the most common form of dwarfism. The drug aims to improve bone growth by targeting a specific protein. Researchers will monitor safety and growth over several years, including children wh…