Daniël van As
- Role: PhD Candidate
Myotonic Dystrophy Type 1 (DM1) is the most common form of muscular dystrophy in adults. It affects people in very different ways, with symptoms and disease progression varying greatly from person to person. This unpredictability creates a strong need for more personalized care. In this PhD project, we aim to better understand why DM1 is so variable. We study both biological and clinical data to explore differences in symptoms, how fast the disease progresses, and how patients respond to treatment. Our main goal is to develop advanced computer models using modern machine learning techniques. These models will combine many types of data and help predict how the disease will develop in each individual. This could improve how doctors guide patients and how treatments are evaluated, leading to more personalized and effective care. In addition, we are researching biomarkers at the RNA and protein level that could serve as future tools to monitor disease changes. These biomarkers may also help as clinical trial endpoints, making it easier and more accurate to test potential therapies.