Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
- Status
- COMPLETED
- Phase
- PHASE3
Clinical Trials Related to BioMarin Pharmaceutical
Related studies linked through entity extraction aliases.
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
- Status
- COMPLETED
- Phase
- PHASE3
Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders
- Status
- COMPLETED
- Phase
- N/A
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
- Status
- TERMINATED
- Phase
- PHASE1/PHASE2
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
- Status
- COMPLETED
- Phase
- PHASE2
Bone Mineral Density in Adults With Hyperphenylalaninemia
- Status
- COMPLETED
- Phase
- N/A
Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
- Status
- COMPLETED
- Phase
- NA
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
- Status
- COMPLETED
- Phase
- PHASE3
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
- Status
- COMPLETED
- Phase
- N/A
The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
- Status
- COMPLETED
- Phase
- N/A
Open-Label Extension Study of Kuvan for Autism
- Status
- COMPLETED
- Phase
- PHASE2/PHASE3
Trial of Kuvan in Lesch-Nyhan Disease
- Status
- WITHDRAWN
- Phase
- PHASE2/PHASE3
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
- Status
- COMPLETED
- Phase
- PHASE2
Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
- Status
- COMPLETED
- Phase
- PHASE2
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
- Status
- COMPLETED
- Phase
- NA
Sapropterin as a Treatment for Autistic Disorder
- Status
- COMPLETED
- Phase
- PHASE2
Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
- Status
- COMPLETED
- Phase
- PHASE3