In addition to identifying a compound that is already in clinical trials for the treatment of ALS (Na-4-phenylbutyrate), our screen also identified other HDAC inhibitors, EZH1/EZH2 inhibitors, HAT activators, and SIRT1 activators that improved cell viability and restored nucleocytoplasmic transport in PR50-expressing cells. The gene discussed is TMPRSS11D; the disease is amyotrophic lateral sclerosis.