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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: 168
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NCT IDTitle
NCT03017677A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis
NCT00272792Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT05580692A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT05734196The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
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
NCT02153255Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
NCT02392793Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT04323098Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT00924703Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
NCT01209819Bone Mineral Density in Adults With Hyperphenylalaninemia
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
NCT01924845BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
NCT06280209A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT01966029BMN 110 Phase 3B in Australian Patients
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT03424018An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT04684940Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT01819727An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT05356377Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
NCT06738017Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT01376908Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
NCT04227080BH4 Responsiveness in PAH Deficiency PKU Patients
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT02055157A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT01415427Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT02156674Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
NCT01275066A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT02537561Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT02576795Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
NCT03370913Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)