Biomarin Pharmaceutical

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is…

This study is for people with Duchenne muscular dystrophy (DMD) who have already taken part in a previous trial of BMN 351. It tests the long-term safety of weekly intravenous infusions of BMN 351 and whether it helps maintain physical function. Only 18 participants will be enrol…

This early-stage trial tests a single oral dose of BMN 349 in 6 adults with a genetic form of alpha-1 antitrypsin deficiency (PiZZ or PiMZ/MASH). The main goal is to check safety and how the drug moves through the body. Participants receive either BMN 349 or a placebo and are clo…

This study tests a rapid drug desensitization (RDD) protocol for adults with phenylketonuria (PKU) who have had allergic reactions to Palynziq. Over one day, patients receive gradually increasing doses to retrain their immune system. The goal is to see if they can safely restart …

This study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese men with severe hemophilia A. The goal is to help their bodies produce their own clotting factor, reducing or stopping the need for regular factor VIII infusions. Participants will be monitored fo…

This study tests a daily injection called BMN 111 in about 70 children with achondroplasia, the most common form of dwarfism. The goal is to see if the drug is safe and helps children grow taller over several years. All participants had already received BMN 111 or a placebo in a …

This study tests a drug called vosoritide in infants with achondroplasia (the most common form of dwarfism) who are at high risk of needing a surgery to relieve pressure on the spinal cord at the base of the skull. The main goal is to see if the drug is safe and if it can help en…

This study tests a drug called BMN 111 in children with achondroplasia, the most common form of dwarfism. The goal is to see if it safely helps them grow taller over several years. About 119 children who completed an earlier study will take the drug and be monitored for growth, d…

This study looks at the long-term safety and effects of a daily injection called BMN 111 in children with achondroplasia, a common form of dwarfism. It includes 30 children who have already taken the drug for two years in a previous study. The goal is to see if the drug remains s…

This study tests a drug called vosoritide to see if it can safely help children with hypochondroplasia grow faster. About 80 children aged 3 to 17 with a confirmed genetic diagnosis and very short stature will receive either the drug or a placebo for one year. The main goal is to…

This study checks the long-term safety and effectiveness of a one-time gene therapy (BMN 270) for people with severe hemophilia A. Participants from earlier BioMarin trials will be followed for many years to see if the treatment continues to work and remains safe. The goal is to …

This study tests an injectable drug called pegvaliase in 55 teenagers (ages 12-17) with phenylketonuria (PKU) who have high blood Phe levels despite diet management. Half get the drug, half stick to diet only. The goal is to see if pegvaliase safely lowers Phe levels and reduces …

This study follows 35 children with CLN2 disease (a rare, severe brain disorder) who are taking cerliponase alfa (Brineura). Researchers will monitor for side effects and serious reactions over time. The goal is to better understand the long-term safety of this enzyme replacement…