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 | ||
|---|---|---|---|
| NCT01826474 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | ||
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | ||
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | ||
| NCT04684940 | Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors | ||
| NCT02156674 | Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome | ||
| NCT07477691 | Immune Modulation During Palynziq® Treatment in Adults (IMPALA) | ||
| NCT02353312 | Rhode Island Diastolic Dysfunction - Heart Failure | ||
| NCT03197766 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT03017677 | A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis | ||
| NCT00272792 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet | ||
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | ||
| NCT02963350 | A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease | ||
| NCT04476862 | Cerliponase Alfa Observational Study in the US | ||
| NCT04560933 | A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A | ||
| NCT01707433 | Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease | ||
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | ||
| NCT04368624 | PKU Skin Stripping | ||
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | ||
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | ||
| NCT01961518 | Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome | ||
| NCT02724228 | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | ||
| NCT01242111 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT04323098 | Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A | ||
| NCT03204370 | Natural History of Atypical Morquio A Disease | ||
| NCT05734196 | The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency | ||
| NCT00838435 | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT05579548 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | ||
| 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 | ||
| NCT00214773 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | ||
| NCT02294877 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | ||
| NCT07473973 | ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | ||
| NCT00423280 | Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study) | ||
| NCT06309979 | A Study to Assess Growth in Children With Idiopathic Short Stature | ||
| NCT05368038 | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | ||
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | ||
| NCT02392793 | Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies | ||
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria | ||
| NCT00884949 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA | ||
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | ||
| NCT02401347 | Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors | ||
| NCT01920828 | Gait Analysis in MPS IVA | ||
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | ||
| NCT01752296 | Psychological Concomitants of Morquio Syndrome (The MAP Study) | ||
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | ||
| NCT02576795 | Gene Therapy Study in Severe Haemophilia A Patients (270-201) |
