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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: 165
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NCT IDTitle
NCT02468570A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
NCT06168201VIrtual STudy in Achondroplasia for the US (VISTA)
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT01733615Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
NCT00852358A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT03694353Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
NCT00048620Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
NCT00789568A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
NCT05813314Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT01858103BMN 110 US Expanded Access Program
NCT03872531Lifetime Impact Study for Achondroplasia
NCT02963350A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT01274026Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT00325962A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT07073014Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT03792451Nutrition Status of Adults Treated With Pegvaliase
NCT01966029BMN 110 Phase 3B in Australian Patients
NCT02576795Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT00272792Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
NCT00884949A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT01957059A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT03583697A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT00214773Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT05270837Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT02365376Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
NCT03820804Nutritional Status in Phenylketonuria
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT06212947A Multicenter Multinational Observational Study of Children With Hypochondroplasia
NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
NCT01425528Study of Kuvan Treatment in Adults With GTPCH Deficiency
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT01435772Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT00532844A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
NCT00332189Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT04476862Cerliponase Alfa Observational Study in the US