ATHN 11: Liver Transplantation Outcomes Study
- Status
- COMPLETED
- Phase
- N/A
Clinical Trials Related to BioMarin Pharmaceutical
Related studies linked through entity extraction aliases.
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE2
Lifetime Impact Study for Achondroplasia
- Status
- COMPLETED
- Phase
- N/A
Retrospective Observational Safety Effectiveness With Kuvan in hpA
- Status
- COMPLETED
- Phase
- N/A
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
- Status
- ENROLLING_BY_INVITATION
- Phase
- N/A
Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
- Status
- COMPLETED
- Phase
- N/A
Nutritional Status in Phenylketonuria
- Status
- COMPLETED
- Phase
- N/A
Nutrition Status of Adults Treated With Pegvaliase
- Status
- COMPLETED
- Phase
- N/A
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
- Status
- COMPLETED
- Phase
- PHASE3
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
- Status
- COMPLETED
- Phase
- PHASE2
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
- Status
- TERMINATED
- Phase
- PHASE1/PHASE2
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
- Status
- COMPLETED
- Phase
- N/A
Lifetime Impact of Achondroplasia Study in Europe-LIAISE
- Status
- COMPLETED
- Phase
- N/A
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
- Status
- COMPLETED
- Phase
- PHASE3
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
- Status
- COMPLETED
- Phase
- PHASE3
Natural History of Atypical Morquio A Disease
- Status
- COMPLETED
- Phase
- N/A
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
- Status
- COMPLETED
- Phase
- PHASE3
Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
- Status
- COMPLETED
- Phase
- N/A
A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
- Status
- UNKNOWN
- Phase
- N/A