Marije B.
- Country
- The Netherlands
- Department
- Pediatric Hematology
Research Overview
- Efficacy and Safety Evaluation of Mitapivat in Pediatric Patients with Pyruvate Kinase Deficiency Not Receiving Regular Transfusions: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study
- Evaluation of Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Adolescents with Paroxysmal Nocturnal Hemoglobinuria: A Phase 2, Open-Label, Single-Arm Study
- Pharmacokinetics, Safety, and Tolerability of Iptacopan Hydrochloride in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria: A Phase 3 Study
Data & Insights
Has run more than one trial at 3 of 12 partner sites.
Specializations
Pediatric Hematology
The investigator specializes in treating rare blood disorders in pediatric populations, with particular focus on Pyruvate Kinase Deficiency management strategies.
- Transfusion-dependent PK deficiency
- Non-transfusion-dependent PK deficiency
- Hemoglobin concentration management
Their research encompasses both transfusion-dependent and independent pediatric patients, exploring novel therapeutic approaches.
Rare Blood Disorders
The investigator conducts research in Paroxysmal Nocturnal Hemoglobinuria, focusing on adolescent patient populations.
- Complement inhibition therapy
- Hemolysis management
- Biological response markers
Their work includes evaluation of therapeutic approaches for rare hematological conditions in young patients.
Therapeutic Innovation
The investigator evaluates novel treatments including mitapivat and pegcetacoplan for rare blood disorders.
- Long-term treatment outcomes
- Safety monitoring
- Pharmacokinetic analysis
Their research focuses on establishing safety and efficacy profiles of innovative therapeutic agents.
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