ACHONDROPLASIA

This study compares a new medicine, BMN 333, to an existing one (vosoritide) in 160 children with achondroplasia, the most common form of dwarfism. The goal is to see which drug works better at increasing height over one year. Children aged 2 to 17 who have not had prior growth t…

This study tests a new medicine called TYRA-300 in children aged 3 to 10 who have achondroplasia (a common form of dwarfism) and still have open growth plates. The goal is to see if the drug is safe and can help them grow faster. About 92 children will take part across multiple c…

This study tests an oral medication called infigratinib in infants and children under 3 years old with achondroplasia, the most common form of dwarfism. The goal is to see if the drug can safely improve growth and body proportions. The trial involves different phases to find the …

This study tests a once-weekly injection called navepegritide in 24 adolescents (ages 12-18) with achondroplasia, the most common form of dwarfism. The goal is to see if it safely increases growth rate over 52 weeks compared to a placebo. The treatment aims to manage the conditio…

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 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—both in those taking the drug VOXZOGO a…

This study follows 260 children with achondroplasia (a common form of dwarfism) for up to 2 years. Researchers measure height, body proportions, medical issues, and quality of life to learn more about how the condition progresses naturally. No treatment is given—the goal is simpl…

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 for tracking the condit…

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,…