Nicole Maria M.
- Country
- Germany
- Department
- pediatric and adolescent medicine
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
- Safety and Tolerability Assessment of Isaralgagene Civaparvovec Gene Therapy in Fabry Disease: A Phase I/II, Multicenter, Open-Label, Dose-Ranging Study
- Phase 3 Randomized Double-Blind Placebo-Controlled Trial of Pariglasgene Brecaparvovec in Glycogen Storage Disease Type Ia Patients
Data & Insights
Has run more than one trial at 24 of 38 partner sites.
Specializations
Rare Genetic Disorders
The investigator specializes in studying and treating Hunter Syndrome, also known as Mucopolysaccharidosis II (MPS II), a rare genetic disorder affecting multiple body systems.
- Genetic Disease Management
- Metabolic Disorders
- Inherited Disease Therapeutics
Dedicated to advancing therapeutic solutions for rare genetic conditions with a particular focus on lysosomal storage disorders.
Neurological Manifestations
Focuses on addressing CNS symptoms associated with genetic disorders, particularly investigating long-term therapeutic approaches for neurological complications in MPS II patients.
- Central Nervous System Disorders
- Neurological Complications
- Brain-targeted Therapies
Explores innovative treatment strategies targeting neurological manifestations in rare genetic disorders.
Long-term Treatment Outcomes
Specializes in evaluating extended treatment protocols and monitoring long-term therapeutic efficacy in patients with rare genetic disorders.
- Treatment Continuation Studies
- Long-term Safety Assessment
- Clinical Outcomes Research
Investigates sustained therapeutic benefits and safety profiles in extended treatment programs.
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