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.
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