Ans V. D. P.
- Country
- The Netherlands
- Department
- Pediatrics
Research Overview
- Long-term Safety and Efficacy Assessment of Intravenous Cipaglucosidase Alfa Co-administered with Oral Miglustat in Adults with Late-onset Pompe Disease
- Efficacy and Safety Evaluation of Avalglucosidase Alfa in Treatment-Naïve Pediatric Patients with Infantile-Onset Pompe Disease (IOPD)
- Evaluation of Avalglucosidase Alfa Efficacy and Safety in Non-Classic Pompe Disease Patients Aged ≥5 Years with Deterioration on Alglucosidase Alfa Therapy
Data & Insights
Has run more than one trial at 2 of 18 partner sites.
Specializations
Pompe Disease Research
The investigator specializes in studying treatment approaches for patients with Pompe disease, also known as glycogen storage disease type II. Their work encompasses both classic and non-classic forms of the condition, with particular attention to enzyme replacement therapies.
- Adult Late Onset Pompe Disease (LOPD)
- Infantile Onset Pompe Disease (IOPD)
- Non-classic Pompe Disease
Their research extends to investigating various therapeutic interventions in different age groups, from infants to adults.
Enzyme Therapy Innovation
The investigator focuses on advancing enzyme replacement therapy approaches, particularly evaluating novel treatments such as avalglucosidase alfa and combination therapies. Their work includes assessment of treatment efficacy in patients whose conditions have shown deterioration on standard treatments.
- Long-term Clinical Effects
- Treatment Safety Profiles
- Therapeutic Optimization
Their research encompasses the development of treatment guidelines and dosing optimization strategies.
Clinical Outcomes
The investigator's research involves comprehensive evaluation of treatment outcomes, including survival rates and ventilator-free survival in infant patients. They also study the economic aspects of treatments and healthcare delivery.
- Patient Survival Metrics
- Quality of Care Assessment
- Health Economic Evaluation
Their work includes analysis of medical care costs and assessment of treatment effectiveness across different patient populations.
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Is something out of date?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.