Movement disorders such as Huntington’s disease (HD), or Machado Joseph’s disease (MJD) are relatively rare, progressive, and yet incurable diseases with devastating consequences for patients and their families. Since they are genetic, it is possible to predict if someone is at risk for developing the disease, but we are still not capable of precisely predicting when the diseases will manifest. Likewise, we lack accurate markers of disease progression, which limits our ability to objectively track patients during disease. Strikingly, we are living exciting times, with the advent of anti-sense oligonucleotide trials, and the identification of novel biomarkers for these diseases.
In this symposium, we will focus on translational aspects of polyglutamine diseases, from the bench to the bedside, and will hear about novel biomarkers for HD and SCA3, alongside with the latest outcomes of the ongoing ASO clinical trial in HD.
This is a field of great interest and expertise in the Portuguese community, bridging from basic scientists to clinical researchers, so we are confident we will attract the participation of many junior scientists who can be selected for oral presentations, thereby enabling a close connections between members of the Portuguese Society for Neuroscience and the Portuguese Society of Movement Disorders.
Progression, biomarkers, and novel therapeutic strategies in Huntington’s disease
Discovering disease-modifying therapies for Machado-Joseph: news from preclinical trials
Static and dynamic ocular motor asymmetries as potential biomarkers in spinocerebellar ataxia type 3
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