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: 169
Selected: 0
| NCT ID | Title | ||
|---|---|---|---|
| NCT02191917 | A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD) | ||
| NCT02392793 | Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies | ||
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | ||
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | ||
| 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 | ||
| NCT03505125 | A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults | ||
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | ||
| NCT02678689 | A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | ||
| NCT03520712 | Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 | ||
| NCT03197766 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT05813314 | Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants | ||
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | ||
| NCT03792451 | Nutrition Status of Adults Treated With Pegvaliase | ||
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | ||
| NCT02221362 | A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease | ||
| NCT06212947 | A Multicenter Multinational Observational Study of Children With Hypochondroplasia | ||
| NCT00048711 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | ||
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) | ||
| NCT01869972 | Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia | ||
| NCT06668805 | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | ||
| NCT06455059 | Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia | ||
| NCT04476862 | Cerliponase Alfa Observational Study in the US | ||
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | ||
| NCT01541397 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | ||
| NCT00423280 | Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study) | ||
| NCT03392974 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg | ||
| NCT01826474 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | ||
| NCT02208661 | Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) | ||
| NCT02365376 | Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions | ||
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT01037309 | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | ||
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | ||
| NCT01889862 | Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU | ||
| NCT03017677 | A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis | ||
| NCT02153255 | Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa | ||
| NCT00214773 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | ||
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | ||
| NCT03424018 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT01141595 | Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders | ||
| NCT02049593 | PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors | ||
| NCT01209819 | Bone Mineral Density in Adults With Hyperphenylalaninemia | ||
| NCT00435331 | 6R-BH4 Pulmonary Arterial Hypertension Study | ||
| NCT03370913 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) | ||
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria |
