New old drug(s) for
spinocerebellar ataxias
Spinocerebellar ataxias (SCAs) are a group
of more than 40 neurodegenerative diseases,
characterized by progressive impairment
of balance, motor coordination and gait.
Ataxin-1 was the first gene identified in an
SCA (SCA1). Purkinje cell degeneration
is a hallmark post mortem feature of most
SCAs, including SCA1, and Purkinje cell
dysfunction is often observed during early
disease stages (Meera et al. 2016). At
present, there is no treatment available
for SCAs, although a paper by Shuvaev
and colleagues in this issue of The Journal
of Physiology identifies a promising new
therapeutic approach for SCA1 using a
drug already approved by the FDA, baclofen
(Shuvaev et al. 2017).
full article (pdf.) http://onlinelibrary.wiley.com/doi/10.1113/JP273149/pdf