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: 170
Selected: 0
| NCT ID | Title | ||
|---|---|---|---|
| NCT04476862 | Cerliponase Alfa Observational Study in the US | ||
| NCT05579548 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | ||
| NCT07477691 | Immune Modulation During Palynziq® Treatment in Adults (IMPALA) | ||
| NCT00225615 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels | ||
| NCT02485899 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease | ||
| NCT06780332 | Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq | ||
| NCT01826474 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | ||
| NCT04227080 | BH4 Responsiveness in PAH Deficiency PKU Patients | ||
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | ||
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | ||
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | ||
| NCT00802893 | Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction | ||
| NCT00688844 | Nutritional and Neurotransmitter Changes in PKU Subjects on BH4 | ||
| NCT00532844 | A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction | ||
| NCT01515956 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT02597881 | Achondroplasia Natural History Multicenter Clinical Study | ||
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | ||
| NCT03864029 | Retrospective Observational Safety Effectiveness With Kuvan in hpA | ||
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease | ||
| NCT04404530 | Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) | ||
| NCT02392793 | Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies | ||
| NCT01889862 | Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU | ||
| NCT01016392 | Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency | ||
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | ||
| NCT03820804 | Nutritional Status in Phenylketonuria | ||
| NCT06224907 | Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A | ||
| NCT01819727 | An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 | ||
| NCT01858103 | BMN 110 US Expanded Access Program | ||
| NCT02156674 | Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome | ||
| NCT01961518 | Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome | ||
| NCT01966029 | BMN 110 Phase 3B in Australian Patients | ||
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | ||
| NCT05356377 | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU | ||
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | ||
| NCT01924026 | Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study | ||
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | ||
| NCT02221362 | A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease | ||
| 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 | ||
| NCT03017677 | A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis | ||
| NCT01753804 | A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. | ||
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | ||
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency | ||
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | ||
| NCT03150069 | Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? | ||
| NCT00332189 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | ||
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | ||
| NCT05734196 | The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency | ||
| NCT00423280 | Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study) |
