This case report documents the neurological, molecular, structural, and electrophysiological reversal of ALS following CBIT2, and plausibly links its therapeutic effect to the induction of the heat shock response targeting misfolded TDP-43, which is based on multiple experimental investigations showing that activation of the heat shock response leads to expression of HSPs, particularly HSP70 and HSP40, which have been shown to facilitate clearance and refolding of misfolded TDP-43 [27,28,29,30,31]. Here, TARDBP is linked to amyotrophic lateral sclerosis.