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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: 170
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NCT IDTitle
NCT00225615A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT01114737Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT00935753Trial of Kuvan in Lesch-Nyhan Disease
NCT03872531Lifetime Impact Study for Achondroplasia
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT00403494A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT04227080BH4 Responsiveness in PAH Deficiency PKU Patients
NCT06738017Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
NCT02049593PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT03370913Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
NCT01541397Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
NCT05579548A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
NCT01806051A Pilot Study on Diurnal Variation
NCT02468570A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
NCT01617070Effects of Kuvan on Melatonin Secretion
NCT07573631An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT00730080Sapropterin in Individuals With Phenylketonuria
NCT04055051ATHN 11: Liver Transplantation Outcomes Study
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT05734196The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
NCT02329769Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT00789568A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
NCT03583697A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
NCT01977820Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
NCT05368038ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
NCT02963350A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
NCT01425528Study of Kuvan Treatment in Adults With GTPCH Deficiency
NCT05580692A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
NCT01961518Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT06280209A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT06224907Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
NCT02485899An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
NCT02055157A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT04368624PKU Skin Stripping
NCT00925054Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia