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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: 169
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NCT IDTitle
NCT01212744Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT01752296Psychological Concomitants of Morquio Syndrome (The MAP Study)
NCT01114737Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
NCT04684940Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT07126262A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
NCT02537561Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors
NCT01387854Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
NCT02353312Rhode Island Diastolic Dysfunction - Heart Failure
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT00104247Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
NCT03204370Natural History of Atypical Morquio A Disease
NCT01415427Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT03424018An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT07441876Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT01141595Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
NCT00850070Sapropterin as a Treatment for Autistic Disorder
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT07073014Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT02392793Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT01617070Effects of Kuvan on Melatonin Secretion
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
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
NCT00048620Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
NCT01858103BMN 110 US Expanded Access Program
NCT01230801Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03792451Nutrition Status of Adults Treated With Pegvaliase
NCT00423280Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study)
NCT01977820Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
NCT02678689A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT03520712Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
NCT00730080Sapropterin in Individuals With Phenylketonuria
NCT06212947A Multicenter Multinational Observational Study of Children With Hypochondroplasia
NCT05314751Learning to Live With Non-severe Haemophilia
NCT01209819Bone Mineral Density in Adults With Hyperphenylalaninemia
NCT01803412A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT00104260Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT00355264Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
NCT00925054Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU