Christina L.
- Country
- Germany
- Department
- Department of Child Neurology, Social Paediatrics and Epileptology
Research Overview
- An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years with Late-onset Pompe Disease
- Long-term Safety and Efficacy Evaluation of Pabinafusp Alfa in Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
- Phase III Evaluation of Pabinafusp Alfa and Idursulfase in Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)
Data & Insights
Has run more than one trial at 10 of 16 partner sites.
Specializations
Rare Genetic Disorders
This investigator specializes in treating rare genetic disorders, with particular emphasis on lysosomal storage diseases. Their research activities demonstrate significant expertise in managing complex inherited metabolic conditions.
- Hunter Syndrome (MPS II)
- Mucopolysaccharidosis Type II
- Late-onset Pompe Disease
The investigator focuses on innovative therapeutic approaches for treating these rare genetic conditions.
Neurological Manifestations
The investigator has developed specific expertise in addressing CNS symptoms associated with rare genetic disorders. Their work encompasses the evaluation of novel treatments targeting neurological manifestations in metabolic disorders.
- Central Nervous System Involvement
- Cognitive Function Assessment
- Long-term Neurological Outcomes
Their research includes evaluation of therapeutic interventions for managing neurological complications in genetic disorders.
Enzyme Replacement Therapies
The investigator explores advanced therapeutic options including enzyme replacement therapy for genetic disorders. Their work involves evaluating novel treatment combinations and therapeutic approaches.
- Innovative Drug Combinations
- Long-term Treatment Efficacy
- Safety Assessment Protocols
The research focuses on developing effective treatment strategies for patients with inherited metabolic disorders.
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