Inaugural lecture: Nicole Wolf

Leukodystrophies, hereditary diseases of the white matter of the brain, are rare. Most often, these conditions lead to neurological decline and often death. Targeted therapies are almost non-existent. This is slowly changing and various treatments are becoming available, sometimes already as a registered medicine, sometimes in research contexts, often in different centers worldwide, and sometimes even as a small n=1 trial for one patient. Nicole Wolf discusses this in her inaugural lecture.
How lessons can be learned from a particular disease course, how the effectiveness of new therapies can be measured in small, often heterogeneous groups and how suitable patients can be selected to demonstrate as quickly as possible whether a treatment works or not, are some questions that Wolf tries to answer. For rare diseases, scientists have long been working across borders. Additional challenges such as COVID19 such as travel restrictions for international patients can provide innovative solutions such as remote monitoring.