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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: 163
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NCT IDTitle
NCT00403494A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT01415427Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01819727An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT05768386A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT00484991Sapropterin Expanded Access Program
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT00355264Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT00943579Open-Label Extension Study of Kuvan for Autism
NCT01376908Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
NCT07073014Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT02963350A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
NCT05813314Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT00787995A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT02576795Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT00924703Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
NCT01858103BMN 110 US Expanded Access Program
NCT05368038ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT03150069Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
NCT01803412A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT06382155A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT01907087A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
NCT03820804Nutritional Status in Phenylketonuria
NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT01435772Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT06168201VIrtual STudy in Achondroplasia for the US (VISTA)
NCT00532844A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
NCT01230801Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT04684940Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT04480567AAV Gene Therapy Study for Subjects with PKU
NCT02153255Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT00789568A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
NCT02329769Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT02597881Achondroplasia Natural History Multicenter Clinical Study
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT03520712Gene Therapy Study in Severe Hemophilia a Patients with Antibodies Against AAV5