Asitri Dashboard Search Clinical Trials
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: 171
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| NCT ID | Title | ||
|---|---|---|---|
| NCT05314751 | Learning to Live With Non-severe Haemophilia | ||
| NCT02294877 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | ||
| NCT00435331 | 6R-BH4 Pulmonary Arterial Hypertension Study | ||
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | ||
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | ||
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | ||
| NCT02153255 | Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa | ||
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency | ||
| NCT02576795 | Gene Therapy Study in Severe Haemophilia A Patients (270-201) | ||
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | ||
| NCT02221362 | A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease | ||
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | ||
| NCT00272792 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet | ||
| NCT02597881 | Achondroplasia Natural History Multicenter Clinical Study | ||
| NCT02583152 | New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis | ||
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | ||
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | ||
| NCT04476862 | Cerliponase Alfa Observational Study in the US | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | ||
| NCT03989947 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | ||
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | ||
| NCT01753804 | A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. | ||
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | ||
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors | ||
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | ||
| NCT06283589 | SEAPORT 1: Study of INZ-701 in Adults With Hemodialysis-Dependent End-Stage Kidney Disease | ||
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | ||
| NCT00067470 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT01617070 | Effects of Kuvan on Melatonin Secretion | ||
| NCT01275066 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT01910649 | A 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 | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | ||
| NCT01752296 | Psychological Concomitants of Morquio Syndrome (The MAP Study) | ||
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | ||
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | ||
| NCT06212947 | A Multicenter Multinational Observational Study of Children With Hypochondroplasia | ||
| NCT01924026 | Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study | ||
| NCT02156674 | Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome | ||
| NCT01541397 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | ||
| NCT00445978 | A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease | ||
| NCT00925054 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU | ||
| NCT00935753 | Trial of Kuvan in Lesch-Nyhan Disease | ||
| NCT04227080 | BH4 Responsiveness in PAH Deficiency PKU Patients | ||
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT01016392 | Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency | ||
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI |
