Spinocerebellar ataxia

T Klockgether, C Mariotti, HL Paulson - Nature reviews Disease primers, 2019 - nature.com
The spinocerebellar ataxias (SCAs) are a genetically heterogeneous group of autosomal
dominantly inherited progressive disorders, the clinical hallmark of which is loss of balance …

Polyglutamine spinocerebellar ataxias—from genes to potential treatments

HL Paulson, VG Shakkottai, HB Clark… - Nature Reviews …, 2017 - nature.com
The dominantly inherited spinocerebellar ataxias (SCAs) are a large and diverse group of
neurodegenerative diseases. The most prevalent SCAs (SCA1, SCA2, SCA3, SCA6 and …

GAA-FGF14 ataxia (SCA27B): phenotypic profile, natural history progression and 4-aminopyridine treatment response

C Wilke, D Pellerin, D Mengel, A Traschütz, MC Danzi… - Brain, 2023 - academic.oup.com
Ataxia due to an autosomal dominant intronic GAA repeat expansion in FGF14 [GAA-FGF14
ataxia, spinocerebellar ataxia 27B (SCA27B)] has recently been identified as one of the …

Spinocerebellar ataxias: prospects and challenges for therapy development

T Ashizawa, G Öz, HL Paulson - Nature Reviews Neurology, 2018 - nature.com
The spinocerebellar ataxias (SCAs) comprise more than 40 autosomal dominant
neurodegenerative disorders that present principally with progressive ataxia. Within the past …

Quantitative gait and balance outcomes for ataxia trials: consensus recommendations by the ataxia global initiative working group on digital-motor biomarkers

W Ilg, S Milne, T Schmitz-Hübsch, L Alcock, L Beichert… - The Cerebellum, 2024 - Springer
With disease-modifying drugs on the horizon for degenerative ataxias, ecologically valid,
finely granulated, digital health measures are highly warranted to augment clinical and …

Autosomal dominant cerebellar ataxias: new genes and progress towards treatments

G Coarelli, M Coutelier, A Durr - The Lancet Neurology, 2023 - thelancet.com
Dominantly inherited spinocerebellar ataxias (SCAs) are associated with phenotypes that
range from pure cerebellar to multisystemic. The list of implicated genes has lengthened in …

Gait variability in spinocerebellar ataxia assessed using wearable inertial sensors

VV Shah, R Rodriguez‐Labrada, FB Horak… - Movement …, 2021 - Wiley Online Library
Background Quantitative assessment of severity of ataxia‐specific gait impairments from
wearable technology could provide sensitive performance outcome measures with high face …

Neurofilaments in spinocerebellar ataxia type 3: blood biomarkers at the preataxic and ataxic stage in humans and mice

C Wilke, E Haas, K Reetz, J Faber… - EMBO molecular …, 2020 - embopress.org
With molecular treatments coming into reach for spinocerebellar ataxia type 3 (SCA 3),
easily accessible, cross‐species validated biomarkers for human and preclinical trials are …

Safety and efficacy of riluzole in spinocerebellar ataxia type 2 in France (ATRIL): a multicentre, randomised, double-blind, placebo-controlled trial

G Coarelli, A Heinzmann, C Ewenczyk… - The Lancet …, 2022 - thelancet.com
Background Riluzole has been reported to be beneficial in patients with cerebellar ataxia;
however, effectiveness in individual subtypes of disease is unclear due to heterogeneity in …

[HTML][HTML] Cerebellar transcranial direct current stimulation in spinocerebellar ataxia type 3: a randomized, double-blind, sham-controlled trial

RP Maas, S Teerenstra, I Toni, T Klockgether… - …, 2022 - Elsevier
Repeated sessions of cerebellar anodal transcranial direct current stimulation (tDCS) have
been suggested to modulate cerebellar-motor cortex (M1) connectivity and decrease ataxia …