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: 168
Selected: 0
| NCT ID | Title | ||
|---|---|---|---|
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 | ||
| NCT02636686 | Extension Study of Drisapersen in DMD Subjects | ||
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | ||
| NCT04368624 | PKU Skin Stripping | ||
| NCT00787995 | A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) | ||
| NCT05314751 | Learning to Live With Non-severe Haemophilia | ||
| NCT01924026 | Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT02365376 | Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions | ||
| NCT04684940 | Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors | ||
| NCT01376908 | Kuvan® in Phenylketonuria Patients Less Than 4 Years Old | ||
| NCT00789568 | A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects | ||
| NCT00964236 | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT06212947 | A Multicenter Multinational Observational Study of Children With Hypochondroplasia | ||
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | ||
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | ||
| NCT02537561 | Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors | ||
| NCT00838435 | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU | ||
| NCT02353312 | Rhode Island Diastolic Dysfunction - Heart Failure | ||
| NCT01920828 | Gait Analysis in MPS IVA | ||
| NCT03520712 | Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 | ||
| NCT01560286 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks | ||
| NCT06309979 | A Study to Assess Growth in Children With Idiopathic Short Stature | ||
| NCT07473973 | ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency | ||
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors | ||
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | ||
| NCT00778206 | PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT00423280 | Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study) | ||
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | ||
| NCT03204370 | Natural History of Atypical Morquio A Disease | ||
| NCT01752296 | Psychological Concomitants of Morquio Syndrome (The MAP Study) | ||
| NCT04476862 | Cerliponase Alfa Observational Study in the US | ||
| NCT01242111 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | ||
| NCT01707433 | Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease | ||
| NCT04800692 | The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication | ||
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria | ||
| NCT05768386 | A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) | ||
| NCT03150069 | Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? | ||
| NCT02156674 | Naglazyme After Allo Transplant for Maroteaux-Lamy 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 | ||
| NCT02583152 | New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis | ||
| NCT05368038 | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | ||
| NCT04055051 | ATHN 11: Liver Transplantation Outcomes Study | ||
| NCT03989947 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | ||
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | ||
| NCT02221362 | A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease | ||
| NCT03856203 | Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase |
