Hannerieke V. D. H.
- Country
- The Netherlands
- Department
- Centre for lysosomal and metabolic disease, Paediatrics
Research Overview
- Long-Term Safety, Tolerability, and Efficacy of Iduronate-2-Sulfatase-Fc Polypeptide in Mucopolysaccharidosis Type II Patients from DNLI-E-0002/0007 Studies
- An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects with Infantile-onset Pompe Disease Aged 0 to <18 Years
- Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Patients with Hunter Syndrome (Mucopolysaccharidosis Type II)
Data & Insights
Has run more than one trial at 10 of 20 partner sites.
Specializations
Rare Genetic Disorders
The investigator specializes in treating complex genetic disorders, with particular emphasis on lysosomal storage diseases affecting pediatric populations.
- Infantile-onset Pompe Disease (IOPD)
- Mucopolysaccharidosis Type II (MPS II)
- Hunter Syndrome
Their work encompasses both treatment-experienced and treatment-naïve patients, focusing on innovative therapeutic approaches.
Pediatric Medicine
The investigator focuses on developing treatments for children and young adults with inherited metabolic disorders, particularly addressing neurodegenerative manifestations.
- Early-onset Disease Management
- Pediatric Enzyme Replacement Therapy
- Long-term Treatment Outcomes
Their research extends from infancy through adolescence, addressing various stages of disease progression.
Therapeutic Innovation
The investigator explores advanced therapeutic solutions, including enzyme replacement therapies and novel drug combinations for rare genetic conditions.
- CNS-targeted Therapies
- Combination Treatment Approaches
- Long-term Safety Monitoring
Their work includes evaluating innovative treatments targeting both systemic and neurological manifestations of genetic disorders.
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