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Project

Evaluating novel radiological and clinical outcome measures in hereditary neuromuscular diseases.

In a considerable number of hereditary neuromuscular diseases new therapeutic possibilities are on the horizon or even already available. To be able to measure an effect of these therapies it is crucial to define accurate and reproducible outcome measures. These optimal outcome measures differ between different neuromuscular diseases, and depend on several parameters such as the rate of disease progression and different course and comorbidities of the diseases. In this PhD research I will study novel and existing clinical and radiological outcome measures in several neuromuscular diseases. I will use magnetic resonance imaging (MRI) of the muscles with determination of the fat fractions (Dixon sequences) and changes in fibrosis/connective tissue (MR elastography) in longitudinal studies for the duration of at least 2 years. The radiological data will be correlated with novel and existing clinical outcome measures. These studies are a necessary basis for future clinical trials with new therapies in hereditary neuromuscular diseases.

Date:1 Jun 2022 →  Today
Keywords:Neuromuscular, Outcome measures, MRI muscles
Disciplines:Neurological and neuromuscular diseases
Project type:PhD project