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The U.S. National Library of Medicine provides a database of clinical trials worldwide. Use the fields below to search the database. Expand your results and select terms or phrases and right-click to search using Google, MalaCards, MedlinePlus, PubMed, or Wikipedia.

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Clinical Trials Search Results

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Count: 168
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NCT IDTitle
NCT00728676Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT00484991Sapropterin Expanded Access Program
NCT00925054Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT01650909The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
NCT01016392Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
NCT01920828Gait Analysis in MPS IVA
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT01819727An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT01541397Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
NCT07473973ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT05270837Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT01617070Effects of Kuvan on Melatonin Secretion
NCT03872531Lifetime Impact Study for Achondroplasia
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT04560933A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT00634660Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
NCT01697319Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
NCT00048711Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
NCT00924703Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
NCT05768386A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
NCT01803412A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT00787995A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT06212947A Multicenter Multinational Observational Study of Children With Hypochondroplasia
NCT00884949A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
NCT00852358A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
NCT02156674Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT00104234Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT02597881Achondroplasia Natural History Multicenter Clinical Study
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT00730080Sapropterin in Individuals With Phenylketonuria
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors