SciencePersonalized RNA therapy reportedly shows early signs of benefit in one ALS patient
A science roundup posted on September 19, 2026, says a patient with a rare CHCHD10 mutation received six doses of a custom treatment over 12 months. It notes that the single-patient study had no control group.
TLDR
A science roundup describes early signs of benefit from a custom antisense oligonucleotide, a personalized RNA therapy, in one ALS patient. According to the post, blood neurofilament light fell by roughly 50%, the patient’s ALS functional score rose from 33 to 36, and predicted vital capacity increased from 48% to 55%. The post notes that these results came from a single-patient study with no control group.
Combined views
147
1 Source, first seen 5d ago
Personalized RNA therapy reportedly shows early signs of benefit in one ALS patient
A science roundup posted on September 19, 2026, says a patient with a rare CHCHD10 mutation received six doses of a custom treatment over 12 months. It notes that the single-patient study had no control group.