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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Count: 164
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| NCT ID | Title | ||
|---|---|---|---|
| NCT01415427 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT03017677 | A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis | ||
| NCT00935753 | Trial of Kuvan in Lesch-Nyhan 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 | ||
| NCT00532844 | A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction | ||
| NCT01242111 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | ||
| NCT01603095 | A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia | ||
| NCT01016392 | Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency | ||
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | ||
| NCT03792451 | Nutrition Status of Adults Treated With Pegvaliase | ||
| NCT02156674 | Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome | ||
| NCT00332189 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | ||
| NCT02597881 | Achondroplasia Natural History Multicenter Clinical Study | ||
| NCT01114737 | Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients | ||
| NCT06224907 | Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A | ||
| NCT02392793 | Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies | ||
| NCT06280209 | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy | ||
| NCT01889862 | Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU | ||
| NCT02191917 | A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD) | ||
| NCT03424018 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria | ||
| NCT02055157 | A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia | ||
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors | ||
| NCT05813314 | Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants | ||
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | ||
| NCT01274026 | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders | ||
| NCT00802893 | Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction | ||
| NCT06305234 | A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) | ||
| NCT00272792 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet | ||
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease | ||
| NCT03197766 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ||
| NCT05314751 | Learning to Live With Non-severe Haemophilia | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT02537561 | Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors | ||
| NCT02576795 | Gene Therapy Study in Severe Haemophilia A Patients (270-201) | ||
| NCT01230801 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease | ||
| NCT01869972 | Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia | ||
| NCT03150069 | Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? | ||
| NCT04055051 | ATHN 11: Liver Transplantation Outcomes Study | ||
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | ||
| NCT02049593 | PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors | ||
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | ||
| NCT01920828 | Gait Analysis in MPS IVA | ||
| NCT05368038 | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | ||
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | ||
| NCT01924845 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) | ||
| NCT06738017 | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | ||
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | ||
| NCT05768386 | A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) | ||
| NCT03449368 | Lifetime Impact of Achondroplasia Study in Europe-LIAISE |
