| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | RECRUITING | PHASE2 | 2025-07-30 | 2028-06-30 | 2028-06-30 |
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | ENROLLING_BY_INVITATION | PHASE3 | 2025-06-20 | 2040-12 | 2040-12 |
| NCT06738017 | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | RECRUITING | PHASE1 | 2025-02-21 | 2025-09 | 2025-09 |
| NCT06780332 | Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq | RECRUITING | PHASE4 | 2025-01 | 2025-11 | 2025-11 |
| NCT06668805 | A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone | RECRUITING | PHASE2 | 2024-11-22 | 2041-09 | 2027-03 |
| NCT06382155 | A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature | RECRUITING | PHASE2 | 2024-10-21 | 2036-12 | 2026-06 |
| NCT06455059 | Interventional Study of Vosoritide for the Treatment of Children with Hypochondroplasia | ENROLLING_BY_INVITATION | PHASE3 | 2024-06-06 | 2026-08-01 | 2026-08-01 |
| NCT06280209 | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy | RECRUITING | PHASE1, PHASE2 | 2024-01-03 | 2026-09-30 | 2026-09-30 |
| NCT06224907 | Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A | ACTIVE_NOT_RECRUITING | PHASE3 | 2023-12-25 | 2029-03 | 2025-03 |
| NCT06138327 | A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria | WITHDRAWN | PHASE1 | 2023-09-26 | 2024-03-25 | 2024-03-25 |
| NCT05813314 | Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants | TERMINATED | PHASE1 | 2023-03-15 | 2023-06-14 | 2023-06-14 |
| NCT05580692 | A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV) | TERMINATED | NA | 2023-03-14 | 2024-07-31 | 2024-07-31 |
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | ACTIVE_NOT_RECRUITING | PHASE3 | 2022-06-17 | 2027-10 | 2025-01-14 |
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | ACTIVE_NOT_RECRUITING | PHASE1, PHASE2 | 2022-02-15 | 2028-11 | 2028-11 |
| NCT04684940 | Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors | ACTIVE_NOT_RECRUITING | PHASE1, PHASE2 | 2020-12-10 | 2029-04 | 2029-04 |
| NCT04323098 | Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A | COMPLETED | PHASE3 | 2020-12-08 | 2025-05-08 | 2023-01-27 |
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | ACTIVE_NOT_RECRUITING | PHASE2 | 2020-10-10 | 2027-12 | 2027-12 |
| NCT04480567 | AAV Gene Therapy Study for Subjects with PKU | ACTIVE_NOT_RECRUITING | PHASE1, PHASE2 | 2020-09-24 | 2027-12 | 2027-12 |
| NCT03989947 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | ACTIVE_NOT_RECRUITING | PHASE2 | 2019-06-12 | 2038-05 | 2038-05 |
| NCT03694353 | Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU | COMPLETED | PHASE3 | 2018-09-13 | 2021-01-13 | 2021-01-13 |
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | COMPLETED | PHASE2 | 2018-06-13 | 2022-01-26 | 2022-01-26 |
| NCT03520712 | Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 | TERMINATED | PHASE1, PHASE2 | 2018-04-24 | 2024-08-07 | 2024-08-07 |
| NCT03392974 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg | COMPLETED | PHASE3 | 2018-03-14 | 2023-06-05 | 2019-05-22 |
| NCT03370913 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) | COMPLETED | PHASE3 | 2017-12-19 | 2024-11-20 | 2020-11-16 |
| NCT03424018 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | ACTIVE_NOT_RECRUITING | PHASE3 | 2017-12-12 | 2031-06 | 2031-06 |
| NCT03197766 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | COMPLETED | PHASE3 | 2016-12-12 | 2019-10-30 | 2019-10-30 |
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | TERMINATED | PHASE2 | 2016-04 | 2016-09 | 2016-09 |
| NCT02724228 | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | ACTIVE_NOT_RECRUITING | PHASE2 | 2016-01-26 | 2028-02 | 2027-12 |
| NCT02678689 | A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | COMPLETED | PHASE2 | 2016-01-22 | 2022-04-20 | 2022-04-20 |
| NCT02576795 | Gene Therapy Study in Severe Haemophilia A Patients (270-201) | COMPLETED | PHASE1, PHASE2 | 2015-09-28 | 2024-02-14 | 2024-02-14 |
| NCT02485899 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease | COMPLETED | PHASE1, PHASE2 | 2015-02 | 2020-12-10 | 2020-12-10 |
| NCT02329769 | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | TERMINATED | PHASE2 | 2014-12 | 2016-08-31 | 2016-07-01 |
| NCT01924845 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) | TERMINATED | PHASE3 | 2014-04 | 2016-09-12 | 2016-09-12 |
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | TERMINATED | PHASE2 | 2014-02 | 2014-11 | 2014-11 |
| NCT02055157 | A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia | COMPLETED | PHASE2 | 2014-01-13 | 2017-10-02 | 2017-10-02 |
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | COMPLETED | PHASE4 | 2013-10 | 2023-01-04 | 2023-01-04 |
| 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 | COMPLETED | PHASE1, PHASE2 | 2013-09 | 2015-11 | 2015-11 |
| NCT01889862 | Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU | COMPLETED | PHASE3 | 2013-07-29 | 2019-02-05 | 2016-01-13 |
| NCT01966029 | BMN 110 Phase 3B in Australian Patients | COMPLETED | PHASE3 | 2013-07 | 2016-12 | 2016-07 |
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | TERMINATED | PHASE1, PHASE2 | 2013-06 | 2016-08-03 | 2016-08-03 |
| NCT01819727 | An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 | COMPLETED | PHASE3 | 2013-05 | 2015-11-25 | 2015-11-25 |
| NCT01803412 | A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects | TERMINATED | PHASE3 | 2013-05-01 | 2016-10-01 | 2016-06-01 |
| NCT01826474 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | TERMINATED | PHASE1, PHASE2 | 2013-01 | 2016-08-31 | 2016-08-31 |
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | TERMINATED | PHASE2 | 2012-08 | 2014-10 | 2014-10 |
| NCT01560286 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks | COMPLETED | PHASE2 | 2012-05 | 2015-07 | 2013-09 |
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | TERMINATED | PHASE2 | 2012-04 | 2014-11 | 2014-11 |
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | COMPLETED | PHASE1 | 2012-02 | 2012-06 | 2012-06 |
| NCT01515956 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) | COMPLETED | PHASE2 | 2011-10 | 2016-02 | 2016-02 |
| NCT01435772 | Extension Study for Patients Who Have Participated in a BMN 701 Study | TERMINATED | PHASE2 | 2011-08-15 | 2016-09-09 | 2016-09-09 |
| NCT01415427 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | COMPLETED | PHASE3 | 2011-07 | 2016-06-16 | 2016-06-16 |
| NCT01376908 | Kuvan® in Phenylketonuria Patients Less Than 4 Years Old | COMPLETED | PHASE3 | 2011-06 | 2017-02-17 | 2014-07 |
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) | COMPLETED | PHASE2 | 2011-03 | 2015-04 | 2015-04 |
| NCT01275066 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | COMPLETED | PHASE3 | 2011-02 | 2012-08 | 2012-08 |
| NCT01230801 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease | COMPLETED | PHASE1, PHASE2 | 2011-01-17 | 2013-03-06 | 2013-03-06 |
| NCT01242111 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | TERMINATED | PHASE1, PHASE2 | 2010-11 | 2014-07 | 2014-07 |
| NCT01114737 | Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients | COMPLETED | PHASE3 | 2010-08 | 2013-03 | 2013-03 |
| NCT00924703 | Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) | COMPLETED | PHASE2 | 2010-01-13 | 2019-01-31 | 2019-01-31 |
| NCT01037309 | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | COMPLETED | PHASE1, PHASE2 | 2009-12 | 2013-10 | 2013-05 |
| NCT00925054 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU | COMPLETED | PHASE2 | 2009-09 | 2015-06 | 2015-06 |
| NCT00884949 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA | COMPLETED | PHASE1, PHASE2 | 2009-04 | 2011-03 | 2011-02 |
| NCT00838435 | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU | COMPLETED | PHASE3 | 2009-02 | 2018-09-14 | 2018-09-14 |
| NCT00789568 | A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects | COMPLETED | PHASE1 | 2008-10 | 2009-10 | 2009-01 |
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | COMPLETED | PHASE1 | 2008-05 | 2009-10 | 2009-04 |
| NCT01910649 | A 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 | TERMINATED | PHASE2 | 2008-03 | 2016-09 | 2016-09 |
| NCT00532844 | A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction | COMPLETED | PHASE2 | 2007-09 | 2009-03 | 2008-11 |
| NCT00445978 | A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease | COMPLETED | PHASE2 | 2007-05 | 2009-06 | 2008-08 |
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease | COMPLETED | PHASE2 | 2006-12 | 2009-01 | 2008-11 |
| NCT00355264 | Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency | COMPLETED | PHASE2 | 2006-08 | 2009-06 | 2009-06 |
| NCT00332189 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | COMPLETED | PHASE3 | 2006-07 | 2009-08 | 2009-08 |
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | COMPLETED | PHASE4 | 2006-05 | 2009-04 | 2009-04 |
| NCT00325962 | A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension | COMPLETED | PHASE2 | 2006-05 | 2008-12 | 2006-12 |
| NCT00272792 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet | COMPLETED | PHASE3 | 2006-02 | 2006-11 | |
| NCT00225615 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels | COMPLETED | PHASE3 | 2005-11 | 2006-06 | |
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | COMPLETED | PHASE3 | 2005-03 | 2006-02 | |
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | COMPLETED | PHASE2 | 2004-12 | 2005-11 | |
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | COMPLETED | PHASE3 | 2004-02 | 2006-10 | 2006-10 |
| NCT00067470 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | COMPLETED | PHASE3 | 2003-09 | 2004-03 | |
| NCT00048711 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | COMPLETED | PHASE2 | 2002-03 | 2006-06 | |
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | COMPLETED | PHASE1 | 2000-09 | 2005-11 | |