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@ARTICLE{Klockgether:140671,
author = {Klockgether, Thomas and Mariotti, Caterina and Paulson,
Henry L},
title = {{S}pinocerebellar ataxia.},
journal = {Nature reviews / Disease Primers},
volume = {5},
number = {1},
issn = {2056-676X},
address = {Basingstoke},
publisher = {Nature Publishing Group},
reportid = {DZNE-2020-06993},
pages = {24},
year = {2019},
abstract = {The spinocerebellar ataxias (SCAs) are a genetically
heterogeneous group of autosomal dominantly inherited
progressive disorders, the clinical hallmark of which is
loss of balance and coordination accompanied by slurred
speech; onset is most often in adult life. Genetically, SCAs
are grouped as repeat expansion SCAs, such as
SCA3/Machado-Joseph disease (MJD), and rare SCAs that are
caused by non-repeat mutations, such as SCA5. Most SCA
mutations cause prominent damage to cerebellar Purkinje
neurons with consecutive cerebellar atrophy, although
Purkinje neurons are only mildly affected in some SCAs.
Furthermore, other parts of the nervous system, such as the
spinal cord, basal ganglia and pontine nuclei in the
brainstem, can be involved. As there is currently no
treatment to slow or halt SCAs (many SCAs lead to premature
death), the clinical care of patients with SCA focuses on
managing the symptoms through physiotherapy, occupational
therapy and speech therapy. Intense research has greatly
expanded our understanding of the pathobiology of many SCAs,
revealing that they occur via interrelated mechanisms
(including proteotoxicity, RNA toxicity and ion channel
dysfunction), and has led to the identification of new
targets for treatment development. However, the development
of effective therapies is hampered by the heterogeneity of
the SCAs; specific therapeutic approaches may be required
for each disease.},
keywords = {Age Factors / Disease Progression / Humans / Mass
Screening: methods / Neuroprotective Agents: therapeutic use
/ Postural Balance: physiology / Riluzole: therapeutic use /
Speech Disorders: etiology / Spinocerebellar Ataxias:
diagnosis / Spinocerebellar Ataxias: epidemiology /
Spinocerebellar Ataxias: therapy / Neuroprotective Agents
(NLM Chemicals) / Riluzole (NLM Chemicals)},
cin = {Patient Studies (Bonn)},
ddc = {610},
cid = {I:(DE-2719)1011101},
pnm = {344 - Clinical and Health Care Research (POF3-344)},
pid = {G:(DE-HGF)POF3-344},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:30975995},
doi = {10.1038/s41572-019-0074-3},
url = {https://pub.dzne.de/record/140671},
}