Clinical Trials for BioMarin Pharmaceutical

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature

A Study to Assess Growth in Children With Idiopathic Short Stature

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

VIrtual STudy in Achondroplasia for the US (VISTA)

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

AAV Gene Therapy Study for Subjects with PKU

Cerliponase Alfa Observational Study in the US

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)