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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: 169
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NCT IDTitle
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT01858103BMN 110 US Expanded Access Program
NCT00787995A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT00325962A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
NCT00445978A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
NCT00104247Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT01924026Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
NCT07477691Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
NCT04560933A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
NCT00838435Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT01016392Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT00935753Trial of Kuvan in Lesch-Nyhan Disease
NCT06382155A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT00355264Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
NCT02678689A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT06668805A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
NCT00332189Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT02576795Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT01415427Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT02597881Achondroplasia Natural History Multicenter Clinical Study
NCT01920828Gait Analysis in MPS IVA
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
NCT00634660Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
NCT04368624PKU Skin Stripping
NCT01209819Bone Mineral Density in Adults With Hyperphenylalaninemia
NCT02724228A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT04055051ATHN 11: Liver Transplantation Outcomes Study
NCT03872531Lifetime Impact Study for Achondroplasia
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT02055157A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT06224907Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT05734196The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
NCT00943579Open-Label Extension Study of Kuvan for Autism
NCT05813314Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT01961518Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
NCT07441876Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia