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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

Query: "AREA[SponsorSearch](BioMarin Pharmaceutical)"
Count: 142
Selected: 0
NCT IDTitle
NCT05270837Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT04684940Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT04560933A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
NCT04480567AAV Gene Therapy Study for Subjects With PKU
NCT04476862Cerliponase Alfa Observational Study in the US
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
NCT04323098Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT03872531Lifetime Impact Study for Achondroplasia
NCT03864029Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT03694353Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
NCT03583697A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT03520712Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
NCT03505125A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
NCT03424018An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03392974Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT03370913Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
NCT03197766A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT02963350A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease
NCT02958202Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
NCT02724228A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT02678689A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT02636686Extension Study of Drisapersen in DMD Subjects
NCT02576795Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT02485899A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
NCT02468570A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
NCT02365376Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
NCT02329769Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
NCT02191917A Study of Repiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
NCT02055157A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT01977820Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
NCT01966029BMN 110 Phase 3B in Australian Patients
NCT01965912Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NCT01957059A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
NCT01924845BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
NCT01910649A 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
NCT01907087A 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
NCT01889862Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT01858103BMN 110 US Expanded Access Program
NCT01826474Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
NCT01819727An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT01803412A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT01697319Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
NCT01609062Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome