Since their first description in the early 2000s, [63] pharmacological chaperones have entered the clinical research stage and subsequent practice for rare conformational diseases [64,65], e.g., tafamidis against transthyretin amyloidosis (ATTR), stabilizing the transthyretin (TTR) tetramer [66,67], migalastat, which assists the proper folding of Fabry disease (FD)-related α-galactosidase (GLA) variants [68], etc. Although pharmacological chaperones show promise for ALS treatment, a phase III trial with arimoclomol did not improve outcomes significantly compared to a placebo [69,70]. This evidence concerns the gene TTR and amyotrophic lateral sclerosis.