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 | ||
|---|---|---|---|
| NCT00355264 | Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | ||
| NCT01275066 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | ||
| NCT07441876 | Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | ||
| NCT06738017 | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | ||
| NCT00532844 | A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction | ||
| NCT02294877 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | ||
| NCT01907087 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease | ||
| NCT01924845 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) | ||
| NCT00924703 | Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) | ||
| NCT05314751 | Learning to Live With Non-severe Haemophilia | ||
| NCT04800692 | The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication | ||
| NCT01806051 | A Pilot Study on Diurnal Variation | ||
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 | ||
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | ||
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | ||
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | ||
| NCT00332189 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | ||
| NCT03792451 | Nutrition Status of Adults Treated With Pegvaliase | ||
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | ||
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | ||
| NCT06668805 | A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | ||
| NCT02636686 | Extension Study of Drisapersen in DMD Subjects | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT02583152 | New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis | ||
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) | ||
| NCT02401347 | Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors | ||
| NCT04560933 | A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A | ||
| NCT03505125 | A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults | ||
| NCT02678689 | A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | ||
| NCT00943579 | Open-Label Extension Study of Kuvan for Autism | ||
| NCT04227080 | BH4 Responsiveness in PAH Deficiency PKU Patients | ||
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | ||
| NCT00884949 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA | ||
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease | ||
| NCT01826474 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | ||
| NCT05368038 | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | ||
| NCT03520712 | Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 | ||
| NCT01415427 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT04476862 | Cerliponase Alfa Observational Study in the US | ||
| NCT04404530 | Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) | ||
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | ||
| NCT03872531 | Lifetime Impact Study for Achondroplasia | ||
| NCT01924026 | Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study | ||
| NCT01274026 | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders | ||
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | ||
| NCT01541397 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | ||
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI |
