Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
- Status
- COMPLETED
- Phase
- N/A
Clinical Trials Related to BioMarin Pharmaceutical
Related studies linked through entity extraction aliases.
A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
- Status
- TERMINATED
- Phase
- N/A
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
- Status
- TERMINATED
- Phase
- NA
Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
- Status
- WITHDRAWN
- Phase
- N/A
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
- Status
- COMPLETED
- Phase
- PHASE2
PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors
- Status
- COMPLETED
- Phase
- PHASE1
Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
- Status
- TERMINATED
- Phase
- PHASE2
BMN 110 Phase 3B in Australian Patients
- Status
- COMPLETED
- Phase
- PHASE3
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
- Status
- COMPLETED
- Phase
- PHASE4
Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
- Status
- COMPLETED
- Phase
- N/A
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
- Status
- TERMINATED
- Phase
- PHASE1/PHASE2
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
- Status
- TERMINATED
- Phase
- PHASE3
Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
- Status
- COMPLETED
- Phase
- N/A
Gait Analysis in MPS IVA
- Status
- COMPLETED
- Phase
- N/A
A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
- Status
- TERMINATED
- Phase
- PHASE2
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
- Status
- COMPLETED
- Phase
- PHASE3
Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
- Status
- COMPLETED
- Phase
- N/A
BMN 110 US Expanded Access Program
- Status
- APPROVED_FOR_MARKETING
- Phase
- N/A
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
- Status
- TERMINATED
- Phase
- PHASE1/PHASE2