Indeed, the PIKFYVE inhibitor apilimod (XXX) that was originally identified as a therapeutic compound in iPSC motor neurons derived from C9ORF72‐ALS patients113 reduces DPRs in C9‐BAC mice that harbour the human C9ORF72 with 100–1000 GGGGCC repeats.114. The gene discussed is C9orf72; the disease is amyotrophic lateral sclerosis.