Achondroplasia

This study tests a daily oral drug called TYRA-300 in 92 children aged 3–10 with achondroplasia (a common form of dwarfism). The goal is to see if it safely increases their growth rate. Researchers will try different doses to find the best one. It's a Phase 2 trial, so it's still…

This study tests a new medicine called BMN 333 against an existing drug (vosoritide) to see if it can safely improve growth in children with achondroplasia, the most common form of dwarfism. About 160 children aged 2 to 17 will take part. The goal is to measure how much they grow…

This study tests an oral drug called infigratinib in infants and children under 3 years old with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth and body proportions. The trial has several phases to find the right dose and then co…

This study tests a weekly injection called navepegritide in 24 teenagers (ages 12-18) with achondroplasia, the most common form of dwarfism. The goal is to see if it safely increases growth rate over one year compared to a placebo. The treatment aims to manage the condition by im…

This study tests a weekly injection called TransCon CNP in infants under 2 years old with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth over 52 weeks. About 72 babies will take part, with some receiving the drug and others a pla…

This study observes about 170 children and adults with achondroplasia in the United States over roughly 5 years. Researchers will collect health records and questionnaires to track growth, medical issues, surgeries, and quality of life. The goal is to better understand the condit…

This study is creating a database of health information from people with achondroplasia, a common form of dwarfism. Researchers will collect growth measurements and surgery records from up to 1,500 patients across several U.S. hospitals. The goal is to answer future questions abo…

This study is creating a large database of genetic and physical information from 3100 children with craniofacial abnormalities, such as craniosynostosis and Pierre Robin sequence. Researchers will analyze this data to understand why these conditions vary so much from person to pe…

This study is for people aged 3 and older with achondroplasia or hypochondroplasia (types of skeletal dysplasia). It uses a wearable device called Syde® to measure limb movements over a year. The goal is to find out which movement measurements are reliable and useful for future r…

This study follows 260 children with achondroplasia (ages 2.5 to 11) for up to 2 years to collect data on growth, medical complications, and quality of life. No treatment is given—it is purely observational to better understand the condition's natural course.