BioMarin
Showing 1 - 25 of 155
Severe Hemophilia A Subjects Who Received BMN 270 inPrior
Enrolling by invitation
- Hemophilia A
-
London, United KingdomRoyal London Hospital, Barts and the London Hemophilia Center
Mar 13, 2023
Learning to Live With Non-severe Haemophilia
Not yet recruiting
- Hemophilia
- Questionnaire and interview
- (no location specified)
Apr 6, 2022
Phenylketonuria Trial in Cleveland (saproterin diHCl, Diet treatment)
Completed
- Phenylketonuria
- saproterin dihydrochloride
- Diet treatment
-
Cleveland, OhioUniversity Hospitals Cleveland Medical Center
Apr 26, 2022
Claudication, Intermittent, Peripheral Artery Disease, Peripheral Vascular Diseases Trial in Worcester (Tetrahydrobiopterin 10
Recruiting
- Claudication, Intermittent
- +2 more
- Tetrahydrobiopterin 10 mg/kg
- +3 more
-
Worcester, MassachusettsUMASS Memorial Healthcare - University Campus
Nov 2, 2022
Metastatic Cancer, Unspecified Adult Solid Tumor Trial in Los Angeles (PARP inhibitor BMN-673, temozolomide, irinotecan HCl)
Completed
- Metastatic Cancer
- Unspecified Adult Solid Tumor
- PARP inhibitor BMN-673
- +4 more
-
Los Angeles, CaliforniaJonsson Comprehensive Cancer Center
Sep 22, 2022
Natural History of Atypical Morquio A Disease
Active, not recruiting
- Mucopolysaccharidosis IV A
- Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
-
Bordeaux, Aquitaine, FranceRheumatology department - Bordeaux University Hospital
Feb 15, 2022
Advanced Breast Cancer, HER2/Neu Negative, Triple-Negative Breast Cancer Trial in Stanford (Talazoparib Tosylate)
Active, not recruiting
- Advanced Breast Cancer
- +2 more
- Talazoparib Tosylate
-
Stanford, CaliforniaStanford University Hospitals and Clinics
Nov 16, 2022
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome Trial in Rome (Aldurazyme (laronidase))
Recruiting
- Mucopolysaccharidosis I
- +3 more
- Aldurazyme (laronidase)
-
Rome, ItalyDipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro
Mar 28, 2022
Nutrition Status of PKU Before and During Treatment With
Recruiting
- Phenylketonurias
-
Boston, MassachusettsBoston Children's Hospital
Aug 3, 2021
PKU Skin Stripping
Completed
- Phenylketonurias
- Skin Tape
- Blood Draw
-
Cincinnati, OhioCincinnati Children's Hospital Medical Center
Jul 26, 2021
Children Diagnosed With CLN2 Disease
Recruiting
- Batten Disease
- +2 more
- CLN2 Treatment
-
Columbus, OhioNationwide Children's Hospital
Aug 2, 2021
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Completed
- Pompe Disease
- Thoracic MRI
- +5 more
-
Gainesville, FloridaUniversity of Florida
Jun 28, 2021
Pregnancy With Morquio Syndrome - What Are Patients'
Completed
- Morquio Disease
- MPS - Mucopolysaccharidosis
- Interview
-
Decatur, GeorgiaEmory University
Jan 15, 2021
Maroteaux-Lamy Syndrome Trial in Minneapolis (Naglazyme®)
Terminated
- Maroteaux-Lamy Syndrome
-
Minneapolis, MinnesotaUniversity of Minnesota Medical Center, Fairview
Dec 22, 2020
Childhood Solid Tumors Trial in Memphis (Talazoparib, Irinotecan, Temozolomide)
Completed
- Childhood Solid Tumors
- Talazoparib
- +4 more
-
Memphis, TennesseeSt. Jude Children's Research Hospital
Feb 8, 2021
ATHN 11: Liver Transplantation Outcomes Study
Recruiting
- Hemophilia A and B
- Liver Transplantation
-
Los Angeles, California
- +14 more
Feb 14, 2022
Hemophilia A, Gene Therapy, Clotting Disorders Trial in Johannesburg, London, Southampton (Valoctocogene Roxaparvovec)
Recruiting
- Hemophilia A
- +3 more
- Valoctocogene Roxaparvovec
-
Seoul, Korea, Republic of
- +8 more
Jan 6, 2023
Severe Haemophilia A Trial in United Kingdom (valoctocogene roxaparvovec)
Active, not recruiting
- Severe Haemophilia A
- valoctocogene roxaparvovec
-
Basingstoke, United Kingdom
- +5 more
Feb 22, 2022
A Prospective Clinical Study of Phenylketonuria (PKU)
Active, not recruiting
- Phenylketonurias
-
Aurora, Colorado
- +13 more
Jan 28, 2022
Hyperoxaluria, Nonalcoholic Fatty Liver Disease, Kidney Stone Trial in United States (BMN 255, Placebo)
Recruiting
- Hyperoxaluria
- +2 more
- BMN 255
- Placebo
-
Birmingham, Alabama
- +6 more
Nov 16, 2023
Achondroplasia Trial in Parkville, London, Sheffield (vosoritide)
Active, not recruiting
- Achondroplasia
- vosoritide
-
Parkville, Victoria, Australia
- +2 more
Dec 20, 2022
Late-onset Pompe Disease Trial in Gainesville, London (Respiratory muscle strength measurements by different techniques.)
Terminated
- Late-onset Pompe Disease
- Respiratory muscle strength measurements by different techniques.
-
Gainesville, Florida
- +1 more
Apr 11, 2022