BioMarin Pharmaceutical
BioMarin Pharmaceutical
Explore linked clinical trials.32 results · Page 1 of 2
Clinical trials
Swipe the table sideways for more columns.
| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, arterial calcification of infancy, autosomal recessive hypophosphatemic rickets, autosomal recessive inherited pseudoxanthoma elasticum, cardiovascular disorder, connective tissue disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, hypophosphatemic rickets, autosomal recessive, 1, skin disorder, vascular disorder, vascular hemostatic disease | Active, not recruiting | Phase 1 | 2026-09-29 | NCT05734196 | |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 / Phase 3 | 2026-09-24 | NCT07441876 | |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | Bone Diseases, Developmental, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Musculoskeletal Abnormalities, Noonan syndrome, Skin and Connective Tissue Diseases, bone disorder, cardiovascular disorder, congenital heart disease, connective tissue disorder, endocrine system disorder, heart disorder, hereditary disease, musculoskeletal system disorder | Active, not recruiting | Phase 2 | 2026-09-23 | NCT06668805 | |
| Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | hypochondroplasia | Enrolling by invitation | Phase 3 | 2026-09-09 | NCT07073014 | |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Ceroid Lipofuscinosis, Neuronal, 2, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Fabry disease, GM1 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis III, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Skin and Connective Tissue Diseases, Sulfatidosis, Wolman disease, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrotendinous xanthomatosis, cerebrovascular disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis, vascular disorder, xanthomatosis | Enrolling by invitation | Not stated | 2026-07-28 | NCT05368038 | |
| An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Enrolling by invitation | Phase 2 | 2026-07-24 | NCT07573631 | |
| Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Active, not recruiting | Phase 3 | 2026-07-21 | NCT05270837 | |
| VIrtual STudy in Achondroplasia for the US (VISTA) | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Not stated | 2026-06-30 | NCT06168201 | |
| Cerliponase Alfa Observational Study in the US | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Active, not recruiting | Not stated | 2026-06-30 | NCT04476862 | |
| A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-06-25 | NCT06280209 | |
| A Study to Assess Growth in Children With Idiopathic Short Stature | Idiopathic Short Stature | Recruiting | Not stated | 2026-06-23 | NCT06309979 | |
| Immune Modulation During Palynziq® Treatment in Adults (IMPALA) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Recruiting | Phase 4 | 2026-06-17 | NCT07477691 | |
| A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | Idiopathic Short Stature | Recruiting | Phase 2 | 2026-05-22 | NCT06382155 | |
| Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorder | Active, not recruiting | Phase 1 | 2026-05-06 | NCT06738017 | |
| The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication | Arterial Occlusive Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, arteriosclerosis disorder, atherosclerosis, cardiovascular disorder, intermittent vascular claudication, peripheral arterial disease, peripheral vascular disease, vascular disorder | Active, not recruiting | Phase 1 | 2026-04-15 | NCT04800692 | |
| Achondroplasia Natural History Multicenter Clinical Study | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Not stated | 2026-04-01 | NCT02597881 | |
| A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | hypochondroplasia | Recruiting | Phase 2 | 2026-03-31 | NCT07126262 | |
| A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Recruiting | Not stated | 2026-03-25 | NCT05813678 | |
| A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Recruiting | Not stated | 2026-03-24 | NCT06305234 | |
| A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases and Pregnancy Complications, Nutritional and Metabolic Diseases, Urogenital Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, maternal phenylketonuria, metabolic disease, nervous system disorder, phenylketonuria, pregnancy disorder | Recruiting | Not stated | 2026-03-24 | NCT05579548 | |
| Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, cardiovascular disorder, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, vascular disorder, vascular hemostatic disease | Active, not recruiting | Phase 3 | 2026-03-19 | NCT06224907 | |
| A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 2 | 2026-03-13 | NCT04554940 | |
| An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 2 | 2026-03-13 | NCT03989947 | |
| An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 3 | 2026-03-13 | NCT03424018 | |
| A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 2 | 2026-03-13 | NCT02724228 |