Biomarin Pharmaceutical

This study tests three doses of vosoritide in 30 children with Noonan syndrome who are still growing but didn't get enough height from growth hormone treatment. The main goal is to see if vosoritide can speed up their growth over 6 months. Researchers will also check for side eff…

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 drug called vosoritide in 100 children with idiopathic short stature (short height with no known cause). The goal is to see if it can safely increase growth rate and height compared to standard growth hormone. Participants will receive either vosoritide, growth…

This study follows 450 people with phenylketonuria (PKU) who are taking or starting the drug Palynziq. Over 10 years, researchers will monitor for allergic reactions and other side effects to better understand the drug's long-term safety. Participants receive their usual treatmen…

This study tests a daily injection called vosoritide in 60 infants and toddlers (0 to 3 years old) with hypochondroplasia, a genetic condition that limits bone growth. The goal is to see if the drug safely improves height and growth rate compared to a placebo over 52 weeks. Parti…

This study tests a new medicine, BMN 351, in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The main goal is to check if the drug is safe and how the body handles it. Participants receive multiple doses through an IV, and researchers wil…

This study observes 300 children with idiopathic short stature (short height with no known cause) to collect growth measurements like height and body mass index over time. It does not provide any treatment but aims to gather baseline data to better understand how these children g…

This study watches pregnant women with PKU who take the drug Palynziq (pegvaliase) to learn how it affects their pregnancy and their baby's health. Researchers will track pregnancy outcomes and infant development for up to 50 participants. The goal is to gather safety information…

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…

This study follows 200 people with PKU who are taking or about to start Palynziq (pegvaliase) for up to 10 years. Researchers will track immune responses, inflammation, and lab results to better understand long-term side effects like allergic reactions and joint pain. The goal is…