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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: 162
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NCT IDTitle
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT01275066A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT02055157A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT02156674Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
NCT03862274Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT01977820Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT04476862Cerliponase Alfa Observational Study in the US
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT03872531Lifetime Impact Study for Achondroplasia
NCT00214773Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT00403494A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT02365376Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT00634660Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
NCT01016392Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
NCT05580692A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
NCT00789568A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
NCT01435772Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT03820804Nutritional Status in Phenylketonuria
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT04480567AAV Gene Therapy Study for Subjects with PKU
NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT02329769Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT01924026Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
NCT05579548A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
NCT00838435Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
NCT00104260Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT01752296Psychological Concomitants of Morquio Syndrome (The MAP Study)
NCT05768386A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT00355264Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT00943579Open-Label Extension Study of Kuvan for Autism
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT05813314Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT06168201VIrtual STudy in Achondroplasia for the US (VISTA)
NCT01230801Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
NCT00787995A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT03150069Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
NCT00924703Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)