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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
NCT00403494A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT01733615Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
NCT03505125A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
NCT01209819Bone Mineral Density in Adults With Hyperphenylalaninemia
NCT03204370Natural History of Atypical Morquio A Disease
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT01707433Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT00730080Sapropterin in Individuals With Phenylketonuria
NCT00802893Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication
NCT04480567AAV Gene Therapy Study for Subjects with PKU
NCT03872531Lifetime Impact Study for Achondroplasia
NCT02537561Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors
NCT00104234Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT02678689A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT03820804Nutritional Status in Phenylketonuria
NCT01114737Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
NCT05314751Learning to Live With Non-severe Haemophilia
NCT05813314Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT01920828Gait Analysis in MPS IVA
NCT00299000A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT00838435Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
NCT01961518Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
NCT01924026Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
NCT02677870The Effectiveness of Kuvan in Amish PKU Patients
NCT04476862Cerliponase Alfa Observational Study in the US
NCT00789568A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT01275066A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT01415427Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT03792451Nutrition Status of Adults Treated With Pegvaliase
NCT02597881Achondroplasia Natural History Multicenter Clinical Study
NCT00104260Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
NCT00634660Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
NCT07126262A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
NCT07477691Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
NCT02156674Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
NCT00728676Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors