Biomarin Pharmaceutical
- Country
- 🇨🇦Canada
- Ownership
- -
- Established
- 1997-01-01
- Employees
- -
- Market Cap
- $17.2B
- Website
- http://www.biomarin.com
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
- First Posted Date
- 2016-03-31
- Last Posted Date
- 2025-01-09
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 30
- Registration Number
- NCT02724228
- Locations
- 🇺🇸
Children's Hospital & Research Center Oakland, Oakland, California, United States
🇺🇸Harbor - UCLA Medical Center, Torrance, California, United States
🇺🇸Ann and Robert H. Lurie Childrens Hospital of Chicago, Chicago, Illinois, United States
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
- Conditions
- CLN2 DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2CLN2 DisorderJansky-Bielschowsky Disease
- Interventions
- Biological: BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)Device: Intracerebroventricular access device
- First Posted Date
- 2016-02-10
- Last Posted Date
- 2025-02-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 14
- Registration Number
- NCT02678689
- Locations
- 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇩🇪Universitaetsklinikum Hamburg-Eppendorf, Hamburg, Germany
🇮🇹Children's Hospital Bambino Gesù,IRCCS, Rome, Piazza, Italy
Extension Study of Drisapersen in DMD Subjects
- Conditions
- Duchenne Muscular Dystrophy
- First Posted Date
- 2015-12-22
- Last Posted Date
- 2018-01-24
- Lead Sponsor
- BioMarin Pharmaceutical
- Registration Number
- NCT02636686
- Locations
- 🇺🇸
Kennedy Krieger Institute, Baltimore, Maryland, United States
🇦🇷IMAI Research, Buenos Aires, Argentina
🇦🇺Royal Children's Hosital, Children's Neuroscience Centre, Parkville, Victoria, Australia
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
- First Posted Date
- 2015-10-15
- Last Posted Date
- 2025-04-10
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 15
- Registration Number
- NCT02576795
- Locations
- 🇬🇧
Queen Elizabeth Hospital Birmingham, Birmingham, United Kingdom
🇬🇧Addenbrooke's Hospital, Cambridge, United Kingdom
🇬🇧St. Thomas' Hospital, London, United Kingdom
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
- Conditions
- CLN2 DiseaseBatten DiseaseCLN2 DisorderJansky-Bielschowsky DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2
- First Posted Date
- 2015-06-30
- Last Posted Date
- 2022-08-24
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 23
- Registration Number
- NCT02485899
- Locations
- 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇩🇪Universitaetsklinikum Hamburg-Eppendorf, Hamburg, Germany
🇮🇹Children's Hospital Bambino Gesù,IRCCS, Rome, Piazza, Italy
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
- Conditions
- Phenylketonuria
- First Posted Date
- 2015-06-11
- Last Posted Date
- 2020-06-04
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 9
- Registration Number
- NCT02468570
- Locations
- 🇺🇸
University of Kentucky Medical Center, Lexington, Kentucky, United States
🇺🇸University of Nebraska Medical Center, Omaha, Nebraska, United States
🇺🇸University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, United States
Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
- Conditions
- Rare Genetic Disorders
- First Posted Date
- 2015-02-18
- Last Posted Date
- 2015-02-18
- Lead Sponsor
- BioMarin Pharmaceutical
- Registration Number
- NCT02365376
- Locations
- 🇺🇸
Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- First Posted Date
- 2015-01-01
- Last Posted Date
- 2017-12-08
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 15
- Registration Number
- NCT02329769
- Locations
- 🇧🇪
UZ Leuven, Leuven, Belgium
🇮🇹S.Anna Hospital, Ferrara, Italy
🇮🇹Policlinico Universitario Agostino Gemelli, Roma, Italy
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
- Conditions
- Mucopolysaccharidosis IV Type AMPS IVAMorquio A Syndrome
- First Posted Date
- 2014-11-19
- Last Posted Date
- 2024-03-05
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 418
- Registration Number
- NCT02294877
- Locations
- 🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸Arkansas Children's Hospital, Little Rock, Arkansas, United States
🇺🇸Children's Hospital of Los Angeles, Los Angeles, California, United States
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
- Conditions
- Late-onset Pompe Patients Untreated or Treated With rhGAA
- First Posted Date
- 2014-08-20
- Last Posted Date
- 2016-10-31
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 30
- Registration Number
- NCT02221362
- Locations
- 🇦🇺
Westmead Hospital, Dept. of Genetic Medicine, Westmead, New South Wales, Australia
🇦🇺Women's and Children's Hospital, Adelaide, South Australia, Australia
🇦🇺Monash Medical Centre, Clayton, Victoria, Australia