Defining SOD1 ALS natural history to guide therapeutic clinical trial design.
Taha BaliWade SelfJingxia LiuTeepu SiddiqueLeo H WangThomas D BirdElena RattiNazem AtassiKevin B BoylanJonathan D GlassNicholas J MaragakisJames B CaressLeo F McCluskeyStanley H AppelJames P WymerSummer GibsonLorne ZinmanTahseen MozaffarBrian CallaghanApril L McVeyJennifer Jockel-BalsarottiPeggy AllredElena R FisherGlenn LopateAlan PestronkMerit E CudkowiczTimothy M MillerPublished in: Journal of neurology, neurosurgery, and psychiatry (2016)
SOD1A4V is an aggressive, but relatively homogeneous form of ALS. These SOD1-specific ALS natural history data will be important for the design and implementation of clinical trials in the ALSSOD1 patient population.