ACHONDROPLASIA

This study tests a weekly injection called navepegritide in 24 adolescents aged 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. Participants will receive either the drug or an …

This study tests an oral drug called infigratinib in infants and children under 3 with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth and body proportions. About 77 children will take either the drug or a placebo, and researchers…

This study tests a new medicine called BMN 333 against an existing drug (vosoritide) to see which works better for helping children with achondroplasia grow taller. About 160 children ages 2 to 17 will take part. The goal is to measure how fast they grow over one year and check f…

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 compared to a placebo. About 72 babies will take part, receiving either the drug or a dummy …

This study tests a new medicine called TYRA-300 in children aged 3 to 10 with achondroplasia (the most common form of dwarfism). The goal is to see if it is safe and can help them grow taller. About 92 children will take part, and the study will look at growth speed and side effe…

This study follows 260 children with achondroplasia (a type of dwarfism) for up to 2 years to collect information on their growth, medical issues, and quality of life. No treatments or drugs are given—it is purely observational. The goal is to better understand the natural course…

This study is creating a database of health information from people with achondroplasia, a bone growth condition. Researchers will collect data from medical records of up to 1,500 patients across several U.S. hospitals. The goal is to better understand growth patterns, surgeries,…

This study aims to find out if a wearable device called Syde® can reliably measure limb movement in people with achondroplasia or hypochondroplasia, two conditions that affect bone growth. About 40 participants aged 3 and older who can walk unassisted will wear the device for rec…

This study follows about 170 people with achondroplasia (a common form of dwarfism) in the United States for up to 5 years. Researchers will collect health records and questionnaires to understand growth, medical issues, and quality of life. The goal is to learn more about the co…