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1998 3
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2008 2
2009 4
2010 1
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2012 4
2013 3
2014 4
2015 4
2016 9
2017 3
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2023 7
2024 4

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Production of Spinocerebellar Ataxia Type 3 Model Mice by Intravenous Injection of AAV-PHP.B Vectors.
Konno A, Shinohara Y, Hirai H. Konno A, et al. Int J Mol Sci. 2024 Jun 29;25(13):7205. doi: 10.3390/ijms25137205. Int J Mol Sci. 2024. PMID: 39000316 Free PMC article.
We aimed to produce a mouse model of spinocerebellar ataxia type 3 (SCA3) using the mouse blood-brain barrier (BBB)-penetrating adeno-associated virus (AAV)-PHP.B. Four-to-five-week-old C57BL/6 mice received injections of high-dose (2.0 10(11) vg/mouse …
We aimed to produce a mouse model of spinocerebellar ataxia type 3 (SCA3) using the mouse blood-brain barrier (BBB)-pen …
Single-Session Cerebellar Transcranial Direct Current Stimulation Improves Postural Stability and Reduces Ataxia Symptoms in Spinocerebellar Ataxia.
Brito R, Fabrício JV, Araujo A, Barreto G, Baltar A, Monte-Silva K. Brito R, et al. Cerebellum. 2024 May 2. doi: 10.1007/s12311-024-01696-9. Online ahead of print. Cerebellum. 2024. PMID: 38693314
Spinocerebellar ataxia (SCA) results in balance and coordination impairment, and current treatments have limited efficacy. ...However, the combined effects of ctDCS and postural training on individuals with spinocerebellar ataxia remain underexplored.
Spinocerebellar ataxia (SCA) results in balance and coordination impairment, and current treatments have limited efficacy. ...
Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C.
Bremova-Ertl T, Ramaswami U, Brands M, Foltan T, Gautschi M, Gissen P, Gowing F, Hahn A, Jones S, Kay R, Kolnikova M, Arash-Kaps L, Marquardt T, Mengel E, Park JH, Reichmannová S, Schneider SA, Sivananthan S, Walterfang M, Wibawa P, Strupp M, Martakis K. Bremova-Ertl T, et al. N Engl J Med. 2024 Feb 1;390(5):421-431. doi: 10.1056/NEJMoa2310151. N Engl J Med. 2024. PMID: 38294974 Clinical Trial.
METHODS: In this double-blind, placebo-controlled, crossover trial, we randomly assigned patients 4 years of age or older with genetically confirmed Niemann-Pick disease type C in a 1:1 ratio to receive NALL for 12 weeks, followed by placebo for 12 weeks, or …
METHODS: In this double-blind, placebo-controlled, crossover trial, we randomly assigned patients 4 years of age or older with …
Efficacy of cerebellar transcranial magnetic stimulation in spinocerebellar ataxia type 3: a randomized, single-blinded, controlled trial.
Shi Y, Zou G, Chen Z, Wan L, Peng L, Peng H, Shen L, Xia K, Qiu R, Tang B, Jiang H. Shi Y, et al. J Neurol. 2023 Nov;270(11):5372-5379. doi: 10.1007/s00415-023-11848-2. Epub 2023 Jul 11. J Neurol. 2023. PMID: 37433893
BACKGROUND: Spinocerebellar ataxia type 3 (SCA3) is the most common subtype of SCA without effective treatment. ...Primary outcomes included the Scale for the Assessment and Rating of Ataxia (SARA) and the International Cooperative Ataxia Rating …
BACKGROUND: Spinocerebellar ataxia type 3 (SCA3) is the most common subtype of SCA without effective treatment. ...Prim …
DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results.
Finkel RS, Day JW, Pascual Pascual SI, Ryan MM, Mercuri E, De Vivo DC, Montes J, Gurgel-Giannetti J, Monine M, Gambino G, Makepeace C, Foster R, Berger Z; DEVOTE Study Group. Finkel RS, et al. J Neuromuscul Dis. 2023;10(5):813-823. doi: 10.3233/JND-221667. J Neuromuscul Dis. 2023. PMID: 37393513 Free PMC article.
OBJECTIVE: Here we describe both the design of DEVOTE (NCT04089566), a 3-part clinical study evaluating safety, tolerability, and efficacy of higher dose of nusinersen, and results from the initial Part A. METHODS: DEVOTE Part A evaluates safety and tolerability of a highe …
OBJECTIVE: Here we describe both the design of DEVOTE (NCT04089566), a 3-part clinical study evaluating safety, tolerability, and eff …
Responsiveness of the Scale for the Assessment and Rating of Ataxia and Natural History in 884 Recessive and Early Onset Ataxia Patients.
Traschütz A, Adarmes-Gómez AD, Anheim M, Baets J, Brais B, Gagnon C, Gburek-Augustat J, Doss S, Hanağası HA, Kamm C, Klivenyi P, Klockgether T, Klopstock T, Minnerop M, Münchau A, Renaud M, Santorelli FM, Schöls L, Thieme A, Vielhaber S, van de Warrenburg BP, Zanni G, Hilgers RD; PREPARE Consortium; Synofzik M. Traschütz A, et al. Ann Neurol. 2023 Sep;94(3):470-485. doi: 10.1002/ana.26712. Epub 2023 Jun 12. Ann Neurol. 2023. PMID: 37243847
OBJECTIVE: The Scale for the Assessment and Rating of Ataxia (SARA) is the most widely applied clinical outcome assessment (COA) for genetic ataxias, but presents metrological and regulatory challenges. ...Whereas sensitivity to change was optimal in mild …
OBJECTIVE: The Scale for the Assessment and Rating of Ataxia (SARA) is the most widely applied clinical outcome assessment (CO …
Effects of cerebellar transcranial alternating current stimulation in cerebellar ataxia: study protocol for a randomised controlled trial.
Liu X, Lin W, Zhang L, Zhang WL, Cheng XP, Lian YH, Li MC, Wang SZ, Chen XY, Gan SR. Liu X, et al. Front Neurosci. 2023 Apr 27;17:1180454. doi: 10.3389/fnins.2023.1180454. eCollection 2023. Front Neurosci. 2023. PMID: 37179566 Free PMC article.
Multiple system atrophy-cerebellar type (MSA-C) and spinocerebellar ataxia type 3 (SCA3) are the most common forms of CA, for which no effective treatment is currently available. ...METHODS/DESIGN: This is a 2-week, triple-blind, randomised, sha …
Multiple system atrophy-cerebellar type (MSA-C) and spinocerebellar ataxia type 3 (SCA3) are the most common for …
Effectiveness of High-Frequency Repetitive Transcranial Magnetic Stimulation in Patients With Spinocerebellar Ataxia Type 3.
Zhou M, Qiu M, Jin Y, Li D, Tao C, Lou D, Hu Z, Wang Y, You Z, Shao Y, Zhu Y, Qu M, Lu X. Zhou M, et al. J ECT. 2024 Mar 1;40(1):15-19. doi: 10.1097/YCT.0000000000000925. Epub 2023 Apr 24. J ECT. 2024. PMID: 37145878 Clinical Trial.
OBJECTIVE: To investigate the effectiveness of high-frequency repetitive transcranial magnetic stimulation (HF-rTMS) on improvement of clinical symptoms in patients with spinocerebellar ataxia type 3 (SCA3). METHODS: Sixteen SCA3 participants diagnosed …
OBJECTIVE: To investigate the effectiveness of high-frequency repetitive transcranial magnetic stimulation (HF-rTMS) on improvement of cl
Gait training with a wearable powered robot during stroke rehabilitation: a randomized parallel-group trial.
Miyagawa D, Matsushima A, Maruyama Y, Mizukami N, Tetsuya M, Hashimoto M, Yoshida K. Miyagawa D, et al. J Neuroeng Rehabil. 2023 Apr 28;20(1):54. doi: 10.1186/s12984-023-01168-x. J Neuroeng Rehabil. 2023. PMID: 37118743 Free PMC article. Clinical Trial.
The clinical trial period was 15 days. The participants performed 10 sessions of gait training (5 times per week) each lasting 30 5 min per day. ...CONCLUSIONS: This novel wearable powered robot may have the potential to improve gait speed of individuals in s …
The clinical trial period was 15 days. The participants performed 10 sessions of gait training (5 times per week) each …
Baseline Clinical and Blood Biomarkers in Patients With Preataxic and Early-Stage Disease Spinocerebellar Ataxia 1 and 3.
Tezenas du Montcel S, Petit E, Olubajo T, Faber J, Lallemant-Dudek P, Bushara K, Perlman S, Subramony SH, Morgan D, Jackman B, Figueroa KP, Pulst SM, Fauret-Amsellem AL, Dufke C, Paulson HL, Öz G, Klockgether T, Durr A, Ashizawa T; READISCA Consortium Collaborators. Tezenas du Montcel S, et al. Neurology. 2023 Apr 25;100(17):e1836-e1848. doi: 10.1212/WNL.0000000000207088. Epub 2023 Feb 16. Neurology. 2023. PMID: 36797067 Free PMC article.
BACKGROUND AND OBJECTIVES: In spinocerebellar ataxia, ataxia onset can be preceded by mild clinical manifestation, cerebellar and/or brainstem alterations, or biomarker modifications. READISCA is a prospective, longitudinal observational study of patie …
BACKGROUND AND OBJECTIVES: In spinocerebellar ataxia, ataxia onset can be preceded by mild clinical manifestatio …
78 results