Peter V. H.
- Country
- The Netherlands
- Department
- Metabolic diseases
Research Overview
- A Phase 1/2 Open-Label Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants with Propionic Acidemia
- A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia
- A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT)
Data & Insights
Has run more than one trial at 6 of 11 partner sites.
Specializations
Metabolic Disorders
The investigator specializes in treating rare metabolic conditions, with particular emphasis on Propionic Acidemia, a severe genetic metabolic disorder affecting protein metabolism.
- Metabolic Disease Management
- Genetic Disorder Treatment
- Novel Therapeutic Approaches
Their research explores innovative therapeutic interventions using mRNA-based treatments for metabolic disorders.
Genetic Diseases
The investigator focuses on treating Mucopolysaccharidosis type I, specifically Hurler Syndrome, a rare genetic condition affecting multiple body systems.
- Lysosomal Storage Disorders
- Gene Therapy Applications
- Stem Cell Transplantation
Their work involves evaluating advanced therapeutic options compared to traditional stem cell transplantation approaches.
Advanced Therapeutics
The investigator specializes in developing and implementing novel treatment strategies for rare genetic conditions, focusing on cell-based therapies and genetic interventions.
- Innovative Treatment Development
- Clinical Efficacy Assessment
- Treatment Safety Evaluation
Their research encompasses comparative studies between emerging therapies and established treatment protocols.
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