| Immune Modulation During Palynziq® Treatment in Adults (IMPALA) (NCT07477691) | BioMarin Pharmaceutical | 2026-05-29 |
| An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy (NCT07573631) | BioMarin Pharmaceutical | 2026-04-20 |
| Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia (NCT07073014) | BioMarin Pharmaceutical | 2025-06-20 |
| Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants (NCT06738017) | BioMarin Pharmaceutical | 2025-02-21 |
| Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq (NCT06780332) | BioMarin Pharmaceutical | 2025-02-07 |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment (NCT06668805) | BioMarin Pharmaceutical | 2024-11-22 |
| A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature (NCT06382155) | BioMarin Pharmaceutical | 2024-10-21 |
| A Study to Assess Growth in Children With Idiopathic Short Stature (NCT06309979) | BioMarin Pharmaceutical | 2024-08-08 |
| Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia (NCT06455059) | BioMarin Pharmaceutical | 2024-06-17 |
| A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) (NCT06305234) | BioMarin Pharmaceutical | 2024-01-20 |
| A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy (NCT06280209) | BioMarin Pharmaceutical | 2024-01-03 |
| Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (NCT06224907) | BioMarin Pharmaceutical | 2023-12-25 |
| A Multicenter Multinational Observational Study of Children With Hypochondroplasia (NCT06212947) | BioMarin Pharmaceutical | 2023-11-27 |
| A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria (NCT06138327) | BioMarin Pharmaceutical | 2023-09-26 |
| Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants (NCT05813314) | BioMarin Pharmaceutical | 2023-03-15 |
| A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV) (NCT05580692) | BioMarin Pharmaceutical | 2023-03-14 |
| VIrtual STudy in Achondroplasia for the US (VISTA) (NCT06168201) | BioMarin Pharmaceutical | 2023-02-21 |
| A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) (NCT05768386) | BioMarin Pharmaceutical | 2023-01-01 |
| A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding (NCT05579548) | BioMarin Pharmaceutical | 2022-11-22 |
| Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria (NCT05270837) | BioMarin Pharmaceutical | 2022-06-17 |
| A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) (NCT05813678) | BioMarin Pharmaceutical | 2022-06-06 |
| A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (NCT05121376) | BioMarin Pharmaceutical | 2022-02-15 |
| Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors (NCT04684940) | BioMarin Pharmaceutical | 2020-12-10 |
| Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (NCT04323098) | BioMarin Pharmaceutical | 2020-12-08 |
| A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia (NCT04554940) | BioMarin Pharmaceutical | 2020-10-10 |
| AAV Gene Therapy Study for Subjects with PKU (NCT04480567) | BioMarin Pharmaceutical | 2020-09-24 |
| A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A (NCT04560933) | BioMarin Pharmaceutical | 2020-08-25 |
| Cerliponase Alfa Observational Study in the US (NCT04476862) | BioMarin Pharmaceutical | 2020-08-19 |
| A Prospective Clinical Study of Phenylketonuria (PKU) (NCT04452513) | BioMarin Pharmaceutical | 2019-10-11 |
| An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia (NCT03989947) | BioMarin Pharmaceutical | 2019-06-12 |
| Lifetime Impact Study for Achondroplasia (NCT03872531) | BioMarin Pharmaceutical | 2019-03-31 |
| Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU (NCT03694353) | BioMarin Pharmaceutical | 2018-09-13 |
| A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia (NCT03583697) | BioMarin Pharmaceutical | 2018-06-13 |
| Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 (NCT03520712) | BioMarin Pharmaceutical | 2018-04-24 |
| A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults (NCT03505125) | BioMarin Pharmaceutical | 2018-03-31 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (NCT03392974) | BioMarin Pharmaceutical | 2018-03-14 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (NCT03370913) | BioMarin Pharmaceutical | 2017-12-19 |
| Lifetime Impact of Achondroplasia Study in Europe-LIAISE (NCT03449368) | BioMarin Pharmaceutical | 2017-12-17 |
| An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia (NCT03424018) | BioMarin Pharmaceutical | 2017-12-12 |
| Retrospective Observational Safety Effectiveness With Kuvan in hpA (NCT03864029) | BioMarin Pharmaceutical | 2017-10-10 |