Biomarin Pharmaceutical

This study is testing the long-term safety and effectiveness of an experimental drug called BMN 111 in children with achondroplasia, the most common form of dwarfism. The main goal is to see if the drug can safely increase the children's annual growth rate over many years. Resear…

This study continues testing a daily injection called BMN 111 in about 70 children with achondroplasia, the most common form of dwarfism. The goal is to see if long-term treatment is safe and helps children grow taller until they reach near-adult height. Researchers will also che…

This study is testing a single-dose gene therapy called valoctocogene roxaparvovec (BMN 270) for Japanese adults with severe hemophilia A. The goal is to see if this one-time intravenous treatment can help the body produce its own blood-clotting factor, reducing or eliminating th…

This study follows children with achondroplasia, a genetic condition that causes short stature, to see how safe and effective a daily injection called BMN 111 is over a longer period. It includes about 30 children who have already been taking the drug for two years in a previous …

This study aims to track the long-term safety and durability of a one-time gene therapy (BMN 270) for severe Hemophilia A over approximately 10 years. It will follow about 172 patients who received the therapy in earlier BioMarin trials. The main goal is to see if the treatment's…

This study tests the safety of daily vosoritide injections in infants with achondroplasia (the most common form of dwarfism) who are at risk of needing spinal decompression surgery. Researchers want to see if the treatment is safe for very young children and whether it affects sk…

This study monitors the long-term safety of cerliponase alfa, an approved treatment for CLN2 disease, a rare and severe childhood genetic disorder. It follows 35 children who are already receiving or planning to start this treatment. Researchers collect safety data during regular…

This study is testing whether a daily injection called vosoritide can safely help children with hypochondroplasia grow taller. About 80 children, aged 3 to 18, will receive either the real drug or a placebo (a dummy injection) for one year to see which group grows faster. The mai…

This study is testing whether daily injections of a medication called pegvaliase are safe and effective for teenagers with PKU, a genetic disorder that makes it hard to process protein. About 55 teens will be randomly assigned to receive either the injections or continue managing…