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 | ||
|---|---|---|---|
| NCT03449368 | Lifetime Impact of Achondroplasia Study in Europe-LIAISE | ||
| NCT05579548 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | ||
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | ||
| NCT06212947 | A Multicenter Multinational Observational Study of Children With Hypochondroplasia | ||
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | ||
| NCT02678689 | A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | ||
| NCT00884949 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA | ||
| NCT00838435 | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU | ||
| NCT03872531 | Lifetime Impact Study for Achondroplasia | ||
| NCT01242111 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT02294877 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | ||
| NCT02485899 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease | ||
| NCT01603095 | A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia | ||
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | ||
| NCT00214773 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | ||
| NCT00935753 | Trial of Kuvan in Lesch-Nyhan Disease | ||
| NCT01415427 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT00532844 | A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction | ||
| NCT02392793 | Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies | ||
| NCT04368624 | PKU Skin Stripping | ||
| NCT02365376 | Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions | ||
| NCT03820804 | Nutritional Status in Phenylketonuria | ||
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | ||
| NCT01435772 | Extension Study for Patients Who Have Participated in a BMN 701 Study | ||
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | ||
| NCT00067470 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | ||
| NCT04800692 | The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication | ||
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | ||
| NCT01924026 | Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study | ||
| NCT05314751 | Learning to Live With Non-severe Haemophilia | ||
| NCT06309979 | A Study to Assess Growth in Children With Idiopathic Short Stature | ||
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) | ||
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | ||
| NCT03856203 | Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase | ||
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT00964236 | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | ||
| NCT05368038 | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | ||
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors | ||
| NCT01037309 | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT02597881 | Achondroplasia Natural History Multicenter Clinical Study | ||
| NCT06224907 | Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A | ||
| NCT01274026 | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders | ||
| NCT00272792 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet | ||
| NCT01560286 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks | ||
| NCT03370913 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) | ||
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency | ||
| NCT02636686 | Extension Study of Drisapersen in DMD Subjects | ||
| NCT00484991 | Sapropterin Expanded Access Program | ||
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria |
