Current and emerging treatment modalities for spinocerebellar ataxias.
Spinocerebellar ataxia
gene therapy
neurodegenerative diseases
neurostimulation
treatment
Journal
Expert review of neurotherapeutics
ISSN: 1744-8360
Titre abrégé: Expert Rev Neurother
Pays: England
ID NLM: 101129944
Informations de publication
Date de publication:
Feb 2022
Feb 2022
Historique:
pubmed:
27
1
2022
medline:
7
4
2022
entrez:
26
1
2022
Statut:
ppublish
Résumé
Spinocerebellar ataxias (SCA) are a group of rare neurodegenerative diseases that dramatically affect the lives of affected individuals and their families. Despite having a clear understanding of SCA's etiology, there are no current symptomatic or neuroprotective treatments approved by the FDA. Research efforts have greatly expanded the possibilities for potential treatments, including both pharmacological and non-pharmacological interventions. Great attention is also being given to novel therapeutics based in gene therapy, neurostimulation, and molecular targeting. This review article will address the current advances in the treatment of SCA and what potential interventions are on the horizon. SCA is a highly complex and multifaceted disease family with the majority of research emphasizing symptomatic pharmacologic therapies. As pre-clinical trials for SCA and clinical trials for other neurodegenerative conditions illuminate the efficacy of disease modifying therapies such as AAV-mediated gene therapy and ASOs, the potential for addressing SCA at the pre-symptomatic stage is increasingly promising.
Identifiants
pubmed: 35081319
doi: 10.1080/14737175.2022.2029703
pmc: PMC9048095
mid: NIHMS1789910
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
101-114Subventions
Organisme : NINDS NIH HHS
ID : R01 NS104423
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS118179
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS124854
Pays : United States
Organisme : NINDS NIH HHS
ID : R03 NS114871
Pays : United States
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