Projects
TOWARDS TRIAL READINESS IN HEREDITARY NEUROMUSCULAR DISEASES: Developing accurate, feasible and non-invasive outcome measures. KU Leuven
To evaluate the effect of a new treatment in clinical trials, we first need an adequate knowledge of the natural history of the disease in question, and a set of outcome measures to evaluate disease progression. Additionally, these outcome measures should be sensitive, reliable, objective, non-invasive and feasible. This thesis aims to improve trial readiness for three hereditary neuromuscular diseases: LGMDR12, BMD and SMA.
In ...
A preclinical study to treat neuromuscular disease caused by mutations in the small heat check protein HSPB8. University of Antwerp
Clinical and molecular characterization of a novel congenital disease; the role of PREPL in regulated secretion and neuromuscular transmission KU Leuven
PREPL (PRolyl EndoPeptidase-Like) is deleted in a recessive metabolic disorder characterized by muscle weakness and growth hormone deficiency. Based on the homology with PREP (PRolyl EndoPeptidase) PREPL was predicted to encode an oligopeptidase. Although activity of the catalytic machinery has been demonstrated, no peptide substrates have been identified so far. Recently, we have achieved a breakthrough in determining the physiological ...
Learning-based computational strategies for multimodal image analysis of neurovascular diseases KU Leuven
Neurovascular diseases such as ischemic stroke are one of the most prevalent causes of disability worldwide. In ischemic stroke, blood flow to a specific region of the brain is hampered by a thrombosis or embolism occluding a cerebral artery. The main treatment strategies of ischemic stroke consist of restoring perfusion through intravenous injection of a thrombolytic drug and/or endovascular thrombectomy, with time from onset to reperfusion ...