Here we provide links to selected resources which may be of use to researchers, clinicians and patients as well as other stakeholders in the neuromuscular and neurodegenerative field.
Among neurodegenerative (NDD) and neuromuscular (NMD) diseases an increasing number of clinical studies aim to define the natural history of these disorders, develop biomarkers, and investigate therapeutic interventions.
Early and preclinical disease stages are currently attracting particular interest. For such studies, generally accepted standards for sampling and storage of biomaterials, clinical and neuropsychological assessment, quantitative performance tests, stance and gait analysis, neuroimaging as well as neurophysiology are highly needed.
Movement Disorders Clinical Practice – 11 FEB 2016
Brigitte K. Paap PhD et al
Hereditary ataxias are a heterogeneous group of degenerative diseases of the cerebellum, brain stem and spinal cord. Patients may present with isolated ataxia or with additional symptoms going beyond cerebellar deficits. There are an increasing number of clinical studies with the goal to define the natural history of these disorders, develop biomarkers and investigate therapeutic interventions. Early and preclinical disease stages are currently of particular interest.