Recent Advances in the Pharmacology of Spinocerebellar Ataxia
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pharmacology".
Deadline for manuscript submissions: closed (30 November 2023) | Viewed by 3655
Special Issue Editors
Interests: gene therapy; polyglutamine diseases; ataxin-2; ataxin-3; neurodegeneration; stress granules
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Spinocerebellar ataxias (SCAs) comprise a group of neurodegenerative diseases, now including more than 40 different pathologies. While heterogeneous in terms of cause, disease characteristics, and even progression, they are all characterized by a main degeneration of the cerebellum, which is accompanied by degenerative alterations in other regions of the nervous system. This cerebellar degeneration translates into a loss of coordination, particularly gait, being this ataxia the most prominent symptom of SCA patients. While individually most of these diseases are rare, as a group they have an important dimension and are a current focus of research. Importantly, all the treatments available are symptomatic, as there is no disease modifying therapy that could stop or delay the disease progression. Therefore, there is an urgent need for new treatments for these incurable diseases.
In this line, this special issue aims to provide an updated view on the advances made in the development of drugs aiming to treat spinocerebellar ataxias, focusing both on new drugs being used in patients both in clinics and in clinical trials, but also providing an updated view on the promising drugs that are being studied in a preclinical setting.
Dr. Clévio Nóbrega
Prof. Dr. Laura Bannach Jardim
Guest Editors
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Keywords
- spinocerebellar ataxias
- ataxia
- pharmacology
- drugs
- therapies
- neurodegeneration
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