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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: 164
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NCT IDTitle
NCT02963350A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
NCT04476862Cerliponase Alfa Observational Study in the US
NCT00625820Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria
NCT01707433Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
NCT01376908Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
NCT01590446A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
NCT01650909The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
NCT06305234A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
NCT03424018An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT01242111A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT04480567AAV Gene Therapy Study for Subjects with PKU
NCT05813314Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT004353316R-BH4 Pulmonary Arterial Hypertension Study
NCT00403494A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT01617070Effects of Kuvan on Melatonin Secretion
NCT01858103BMN 110 US Expanded Access Program
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT00325962A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT06738017Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT01966029BMN 110 Phase 3B in Australian Patients
NCT00852358A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
NCT01920828Gait Analysis in MPS IVA
NCT04227080BH4 Responsiveness in PAH Deficiency PKU Patients
NCT00787995A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT02597881Achondroplasia Natural History Multicenter Clinical Study
NCT03505125A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT00924703Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
NCT03204370Natural History of Atypical Morquio A Disease
NCT02208661Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
NCT01541397Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
NCT02537561Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Advanced Solid Tumors
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT06224907Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT00214773Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT01609062Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
NCT00067470Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
NCT01037309Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT06138327A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
NCT00048711Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
NCT00943579Open-Label Extension Study of Kuvan for Autism
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT02191917A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT01733615Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA