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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
NCT00214773Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT03204370Natural History of Atypical Morquio A Disease
NCT01387854Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
NCT01609062Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
NCT01961518Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT00299000A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
NCT04368624PKU Skin Stripping
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT02678689A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT06382155A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
NCT01376908Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
NCT00827762Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT06305234A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
NCT01425528Study of Kuvan Treatment in Adults With GTPCH Deficiency
NCT03150069Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
NCT01435772Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT06280209A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT00104260Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
NCT01541397Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
NCT01560286A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT00048711Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT05314751Learning to Live With Non-severe Haemophilia
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
NCT00943579Open-Label Extension Study of Kuvan for Autism
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
NCT00048620Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
NCT01957059A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
NCT05356377Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
NCT01752296Psychological Concomitants of Morquio Syndrome (The MAP Study)
NCT01275066A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT00403494A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT00104234Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT06738017Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT01910649A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
NCT03505125A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT06780332Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT04800692The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication