RNA-targeted therapy improves patient with rare ALS mutation after a year

A man with a rare CHCHD10-linked ALS mutation received an antisense oligonucleotide RNA therapy; a year later his motor and neurological scores improved, breathing function remained stable, and neurofilament-light levels returned to normal, with no serious side effects, suggesting mutation-targeted RNA therapies could extend to other ALS patients—though more people and longer follow-up are needed.