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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: 164
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NCT IDTitle
NCT03204370Natural History of Atypical Morquio A Disease
NCT03424018An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT01435772Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT05356377Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT01858103BMN 110 US Expanded Access Program
NCT05768386A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT03792451Nutrition Status of Adults Treated With Pegvaliase
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT06168201VIrtual STudy in Achondroplasia for the US (VISTA)
NCT01209819Bone Mineral Density in Adults With Hyperphenylalaninemia
NCT02485899An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT01803412A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT05579548A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
NCT02401347Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors
NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT00532844A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
NCT02576795Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
NCT01697319Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT01275066A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01920828Gait Analysis in MPS IVA
NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT04055051ATHN 11: Liver Transplantation Outcomes Study
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT01752296Psychological Concomitants of Morquio Syndrome (The MAP Study)
NCT03862274Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
NCT06382155A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
NCT01230801Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT00214773Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT00730080Sapropterin in Individuals With Phenylketonuria
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
NCT03150069Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
NCT01650909The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
NCT06212947A Multicenter Multinational Observational Study of Children With Hypochondroplasia