Eugen M.
- Country
- Germany
- Department
- SphinCS GmbH
Research Overview
- Phase 1b/2a Open‑Label Study Assessing Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous DNL952 in Adults with Late‑Onset Pompe Disease
- 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 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
Data & Insights
Has run more than one trial at 36 of 72 partner sites.
Specializations
Lysosomal Storage Disorders
This investigator specializes in treating various forms of lysosomal storage disorders, with particular emphasis on Pompe disease, including both infantile-onset and late-onset variants.
- Infantile-onset Pompe Disease Management
- Late-onset Pompe Disease Therapeutics
- Enzyme Replacement Therapy Optimization
The investigator works with novel therapeutic approaches including cipaglucosidase alfa and miglustat combinations.
Neurodegenerative Disorders
Focus on rare genetic conditions affecting the central nervous system, particularly Niemann-Pick disease and GM1/GM2 gangliosidoses.
- Niemann-Pick Type C Treatment
- Gangliosidosis Management
- Ataxia Treatment Approaches
Special interest in developing treatments for late-infantile and juvenile forms of these conditions.
Complex Genetic Disorders
Expertise in treating Mucopolysaccharidosis Type II (Hunter Syndrome) and Gaucher disease, with focus on both neuronopathic and non-neuronopathic manifestations.
- Hunter Syndrome Treatment
- Gaucher Disease Management
- CNS-targeted Therapeutics
Implements innovative approaches in enzyme replacement therapy and substrate reduction therapy.
Related Investigators
Researchers in similar fields or departments
Want to know more about this investigator?
We can share extended profile information and contact details.
Is something out of date?
Tell us what needs correcting and we'll update this profile.