Naoki Atsuta

TL;DR: The primary efficacy outcome was the change in ALSFRS-R score from the baseline to 24 weeks (or at discontinuation if this was after the third cycle) after randomisation and the least-squares mean difference between groups was 2·49 (SE 0·76, 95% CI 0·99-3·98) in favour of edaravone.

TL;DR: Investigating the natural course of SBMA as assessed by nine activities of daily living (ADL) milestones in 223 Japanese SBMA patients found the levels of serum testosterone, an important triggering factor for polyglutamine-mediated motoneuron degeneration, were maintained at relatively high levels even at advanced ages.

TL;DR: iPSC-derived motor neurons from over 30 heterogeneous sporadic ALS cases exhibit pathologies correlated with clinical disease progression, are more similar to FUS/TDP-43 familial ALS than SOD1-ALS and are corrected by repurposing of ropinirole.

TL;DR: It is shown that TDP‐43 and FUS/TLS localize in nuclear Gems through an association with SMN, and that all three proteins function in spliceosome maintenance, indicating that a profound loss of spliceOSome integrity is a critical mechanism common to neurodegeneration in ALS and SMA and may explain cell‐type specific vulnerability of motor neurons.

TL;DR: This study is aimed at evaluating the efficacy and safety of androgen deprivation by leuprorelin acetate in patients with SBMA.