Biomarin Pharmaceutical

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

This study tests three doses of vosoritide in 30 children with Noonan syndrome who are still short despite growth hormone treatment. The main goal is to see if vosoritide can speed up growth over 6 months. Researchers will also monitor safety and long-term effects on height.

This study follows 450 people with phenylketonuria (PKU) who are taking or starting Palynziq injections. Over 10 years, researchers will track side effects like allergic reactions and joint pain to better understand the drug's long-term safety. The goal is to improve real-world u…

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 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 human growth hormone. Participants will receive either vosoritide or standar…

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 observes 300 children with idiopathic short stature (ISS) to collect growth measurements and other health data. It aims to better understand natural growth patterns and factors that may affect growth. No treatment is given; participants simply attend regular check-ups …

This study follows 200 people with PKU who are already taking or about to start pegvaliase. Over 10 years, researchers will monitor immune responses and lab results to better understand long-term safety. The goal is to learn, not to test a new treatment.

This study follows 50 pregnant women with PKU who have taken the drug Palynziq (pegvaliase) to see how it affects their pregnancy and their baby's health. Researchers will track outcomes like miscarriage, birth defects, and infant development. The goal is to gather safety informa…