Biomarin Pharmaceutical
- Country
- đ¨đŚCanada
- 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
- đşđ¸
Uncommon Cures, Chevy Chase, Maryland, United States
Study of BMN 349 Single Dose in (PiZZ) and (PiMZ) Adult Participants
- First Posted Date
- 2024-12-17
- Last Posted Date
- 2025-03-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 12
- Registration Number
- NCT06738017
- Locations
- đşđ¸
University of California, San Diego, San Diego, California, United States
đşđ¸Saint Louis University, Saint Louis, Missouri, United States
đşđ¸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
- đşđ¸
Children's Hospital Colorado, Aurora, Colorado, United States
đşđ¸Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children), Wilmington, Delaware, United States
đşđ¸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
- đşđ¸
Nemours Alfred I. DuPont Hospital for Children, Wilmington, Delaware, United States
đşđ¸Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
đşđ¸Medical College of Wisconsin, Milwaukee, Wisconsin, United States
A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
- Conditions
- Idiopathic Short Stature
- Interventions
- First Posted Date
- 2024-04-24
- Last Posted Date
- 2025-01-20
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 100
- Registration Number
- NCT06382155
- Locations
- đşđ¸
Centricity Research, Columbus, Georgia, United States
đşđ¸Nemours Children's Health System - Corporate Headquarters, Pensacola, Florida, United States
đşđ¸Rocky Mountain Clinical Research - Idaho Falls, Idaho Falls, Idaho, United States
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
- đşđ¸
Center Of Excellence in Diabetes and Endocrinology, Sacramento, California, United States
đşđ¸University of California San Francisco (UCSF), San Francisco, California, United States
đşđ¸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
- đşđ¸
UT Southwestern Medical Center, Dallas, Texas, United States
đşđ¸Northwestern University, Chicago, Illinois, United States
đşđ¸University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, 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
- 2024-12-05
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 18
- Registration Number
- NCT06280209
- Locations
- đłđą
Leids Universitair Medisch Centrum, Leiden, Netherlands
đšđˇYeditepe University Kosuyolu Hospital, Istanbul, Turkey
đŹđ§Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom
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
- đŻđľ
Asahikawa Medical University Hospital, Hokkaido, Asahikawa, Japan
đŻđľNagoya University Hospital, Aichi, Nagoya, Japan
đŻđľTokyo Medical University Hospital, Tokyo, Shinjuku-ku, 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
- đŻđľ
Institute of Science Tokyo Hospital, Tokyo, Japan
đşđ¸Phoenix Children's Hospital, Phoenix, Arizona, United States
đşđ¸UCSF Benioff Children's Hospital, Oakland, California, United States