Natural history of Krabbe disease - a nationwide study in Germany using clinical and MRI data.
Sarah Isabel KriegIngeborg Kraegeloh-MannSamuel GroeschelStefanie Beck-WödlRalf A HusainLudger SchölsChristiane KehrerPublished in: Orphanet journal of rare diseases (2020)
This is the first description of the natural history of Krabbe disease in a larger European cohort using developmental, clinical and MRI data. We would like to highlight the very different clinical and MRI characteristics of the later onset forms. These data are important for counselling affected patients and families and may serve as a basis for future treatment trials.