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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
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT02537561Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors
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
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT04480567AAV Gene Therapy Study for Subjects with PKU
NCT06280209A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT00925054Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT06668805A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
NCT06738017Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT07477691Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT02156674Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
NCT00048711Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
NCT01858103BMN 110 US Expanded Access Program
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT02724228A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT01016392Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
NCT01752296Psychological Concomitants of Morquio Syndrome (The MAP Study)
NCT04368624PKU Skin Stripping
NCT01425528Study of Kuvan Treatment in Adults With GTPCH Deficiency
NCT01037309Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT02678689A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT06305234A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT00214773Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT03204370Natural History of Atypical Morquio A Disease
NCT05270837Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT00850070Sapropterin as a Treatment for Autistic Disorder
NCT00067470Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
NCT01415427Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01977820Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
NCT04476862Cerliponase Alfa Observational Study in the US
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
NCT06382155A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
NCT02153255Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa