Biomarin Pharmaceutical
- Country
- 🇨🇦Canada
- Ownership
- -
- Established
- 1997-01-01
- Employees
- -
- Market Cap
- $17.2B
- Website
- http://www.biomarin.com
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
- Conditions
- PhenylketonuriaPKU
- Interventions
- Drug: RDD to Palynziq
- First Posted Date
- 2025-01-17
- Last Posted Date
- 2025-01-23
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 10
- Registration Number
- NCT06780332
- Locations
- 🇺🇸
Uncommon Cures, Chevy Chase, Maryland, United States
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
- First Posted Date
- 2024-12-17
- Last Posted Date
- 2025-07-03
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 12
- Registration Number
- NCT06738017
- Locations
- 🇺🇸
University of California, San Diego, San Diego, California, United States
🇺🇸Saint Louis University, St. Louis, Missouri, United States
🇺🇸Medpace Clinical Pharmacology Unit, Cincinnati, Ohio, United States
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
- Conditions
- Short Stature Homeobox- Containing Gene SHOX DeficiencyNoonan SyndromeTurner Syndrome
- Interventions
- First Posted Date
- 2024-10-31
- Last Posted Date
- 2025-05-13
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 72
- Registration Number
- NCT06668805
- Locations
- 🇺🇸
Children's Hospital Colorado, Aurora, Colorado, United States
🇺🇸Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children), Wilmington, Delaware, United States
🇺🇸Nicklaus Children's Hospital, Miami, Florida, United States
Interventional Study of Vosoritide for the Treatment of Children with Hypochondroplasia
- First Posted Date
- 2024-06-12
- Last Posted Date
- 2025-03-19
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 80
- Registration Number
- NCT06455059
- Locations
- 🇺🇸
Nemours Alfred I. DuPont Hospital for Children, Wilmington, Delaware, United States
🇺🇸Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸Medical College of Wisconsin, Milwaukee, Wisconsin, United States
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
- Conditions
- Idiopathic Short Stature (ISS)
- Interventions
- First Posted Date
- 2025-06-24
- Last Posted Date
- 2025-06-24
- Lead Sponsor
- Biomarin Pharmaceutical Inc.
- Target Recruit Count
- 20
- Registration Number
- 2024-520137-74-00
- Locations
- 🇫🇷
Centre Hospitalier Universitaire De Montpellier, Montpellier, France
🇫🇷Centre Hospitalier Regional De Marseille, Marseille, France
🇫🇷Centre Hospitalier Universitaire D'Angers, Angers, France
A Study to Assess Growth in Children With Idiopathic Short Stature
- Conditions
- Idiopathic Short Stature
- First Posted Date
- 2024-03-13
- Last Posted Date
- 2025-04-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 300
- Registration Number
- NCT06309979
- Locations
- 🇺🇸
Center Of Excellence in Diabetes and Endocrinology, Sacramento, California, United States
🇺🇸University of California San Francisco (UCSF), San Francisco, California, United States
🇺🇸Nicklaus Children's Hospital, Miami, Florida, United States
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
- Conditions
- Phenylketonuria (PKU)
- First Posted Date
- 2024-03-12
- Last Posted Date
- 2024-08-26
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 200
- Registration Number
- NCT06305234
- Locations
- 🇺🇸
University of Colorado, Aurora, Colorado, United States
🇺🇸Northwestern University, Chicago, Illinois, United States
🇺🇸Tulane University School of Medicine, New Orleans, Louisiana, United States
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
- First Posted Date
- 2024-02-28
- Last Posted Date
- 2025-06-19
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 18
- Registration Number
- NCT06280209
- Locations
- 🇮🇹
Fondazione Serena Onlus - Centro Clinico NeMO Milano, Milan, Italy
🇮🇹UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore, Rome, Italy
🇳🇱Leids Universitair Medisch Centrum, Leiden, Netherlands
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
- Conditions
- Hemophilia A
- Interventions
- Biological: Valoctocogene roxaparvovec
- First Posted Date
- 2024-01-25
- Last Posted Date
- 2024-05-07
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 6
- Registration Number
- NCT06224907
- Locations
- 🇯🇵
Asahikawa Medical University Hospital, Hokkaido, Asahikawa, Japan
🇯🇵Saitama Medical University Hospital, Saitama, Iruma-gun, Japan
🇯🇵Nagoya University Hospital, Aichi, Nagoya, Japan
A Multicenter Multinational Observational Study of Children With Hypochondroplasia
- Conditions
- Hypochondroplasia
- First Posted Date
- 2024-01-19
- Last Posted Date
- 2025-04-03
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 400
- Registration Number
- NCT06212947
- Locations
- 🇯🇵
Institute of Science Tokyo Hospital, Tokyo, Japan
🇺🇸Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸Cedars-Sinai Medical Center, Los Angeles, California, United States