Moving Towards Therapy in SCA1: Insights from Molecular Mechanisms, Identification of Novel Targets, and Planning for Human Trials
Author:
Funder
National Ataxia Foundation
NIH
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Neurology (clinical),Pharmacology
Link
http://link.springer.com/content/pdf/10.1007/s13311-019-00763-y.pdf
Reference108 articles.
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3. Ashizawa T, Figueroa KP, Perlman SL et al. Clinical characteristics of patients with spinocerebellar ataxias 1, 2, 3 and 6 in the US; a prospective observational study. Orphanet J Rare Dis, 8, 177 (2013).
4. Jacobi H, du Montcel ST, Bauer P et al. Long-term disease progression in spinocerebellar ataxia types 1, 2, 3, and 6: a longitudinal cohort study. Lancet Neurol, 14(11), 1101–1108 (2015).
5. Robitaille Y, Schut L, Kish SJ. Structural and immunocytochemical features of olivopontocerebellar atrophy caused by the spinocerebellar ataxia type 1 (SCA-1) mutation define a unique phenotype. Acta Neuropathol, 90(6), 572–581 (1995).
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