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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: 167
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NCT IDTitle
NCT01819727An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT04560933A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
NCT00332189Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT00048711Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT03820804Nutritional Status in Phenylketonuria
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
NCT01961518Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT02678689A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT01425528Study of Kuvan Treatment in Adults With GTPCH Deficiency
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT06668805A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
NCT02329769Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT03792451Nutrition Status of Adults Treated With Pegvaliase
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT06738017Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT00634660Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
NCT07441876A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT01697319Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
NCT04480567AAV Gene Therapy Study for Subjects with PKU
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT03872531Lifetime Impact Study for Achondroplasia
NCT00532844A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
NCT02468570A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
NCT00943579Open-Label Extension Study of Kuvan for Autism
NCT04684940Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT01733615Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
NCT01752296Psychological Concomitants of Morquio Syndrome (The MAP Study)
NCT05270837Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT05314751Learning to Live With Non-severe Haemophilia
NCT01858103BMN 110 US Expanded Access Program
NCT01806051A Pilot Study on Diurnal Variation
NCT01435772Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT03017677A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase