A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
- Status
- ENROLLING_BY_INVITATION
- Phase
- N/A
Clinical Trials Related to BioMarin Pharmaceutical
Related studies linked through entity extraction aliases.
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
- Status
- RECRUITING
- Phase
- PHASE1
A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
- Status
- TERMINATED
- Phase
- NA
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
- Status
- RECRUITING
- Phase
- N/A
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
- Status
- ENROLLING_BY_INVITATION
- Phase
- N/A
Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
- Status
- COMPLETED
- Phase
- N/A
Learning to Live With Non-severe Haemophilia
- Status
- COMPLETED
- Phase
- N/A
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1/PHASE2
The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1/PHASE2
A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
- Status
- COMPLETED
- Phase
- N/A
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE2
AAV Gene Therapy Study for Subjects with PKU
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1/PHASE2
Cerliponase Alfa Observational Study in the US
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- N/A
A Prospective Clinical Study of Phenylketonuria (PKU)
- Status
- COMPLETED
- Phase
- N/A
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
- Status
- RECRUITING
- Phase
- N/A
PKU Skin Stripping
- Status
- COMPLETED
- Phase
- N/A
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
- Status
- COMPLETED
- Phase
- PHASE3
BH4 Responsiveness in PAH Deficiency PKU Patients
- Status
- UNKNOWN
- Phase
- PHASE4