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 | ||
|---|---|---|---|
| NCT06780332 | Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq | ||
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | ||
| NCT05368038 | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | ||
| NCT01707433 | Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease | ||
| NCT00924703 | Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) | ||
| NCT07573631 | An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy | ||
| NCT01966029 | BMN 110 Phase 3B in Australian Patients | ||
| NCT00225615 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels | ||
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | ||
| NCT01650909 | The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU | ||
| NCT02392793 | Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refractory or Recurrent Solid Malignancies | ||
| NCT06305234 | A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) | ||
| NCT00048711 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | ||
| NCT02537561 | Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors | ||
| NCT03449368 | Lifetime Impact of Achondroplasia Study in Europe-LIAISE | ||
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | ||
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease | ||
| NCT06280209 | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy | ||
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | ||
| NCT02365376 | Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions | ||
| NCT03392974 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg | ||
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | ||
| NCT00964236 | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | ||
| NCT05768386 | A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) | ||
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | ||
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency | ||
| NCT03694353 | Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU | ||
| NCT04480567 | AAV Gene Therapy Study for Subjects with PKU | ||
| NCT00778206 | PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry | ||
| NCT05580692 | A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV) | ||
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | ||
| NCT04684940 | Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors | ||
| NCT02678689 | A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | ||
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | ||
| NCT05356377 | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU | ||
| NCT00325962 | A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension | ||
| NCT02485899 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease | ||
| NCT02294877 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | ||
| NCT01560286 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks | ||
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | ||
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | ||
| NCT04227080 | BH4 Responsiveness in PAH Deficiency PKU Patients | ||
| NCT03505125 | A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults | ||
| NCT01603095 | A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia | ||
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | ||
| NCT02049593 | PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating Patients With Locally Advanced or Metastatic Solid Tumors | ||
| NCT00852358 | A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I | ||
| NCT01753804 | A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. | ||
| NCT02401347 | Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer and Other Solid Tumors | ||
| NCT05813678 | A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) |
