Researchers at Mayo Clinic have reported early results from a personalised therapy designed for a single patient with a rare genetic form of ALS. The bespoke antisense drug targeted his CHCHD10 gene mutation.

The patient received six spinal injections between April 2024 and April 2025. His blood levels of neurofilament light chain, a marker of nerve damage, fell by about 50 per cent to the normal range.

His score on the revised ALS functional rating scale rose from 33 to 36 over the year. Measures of breathing and cognition stayed stable or improved slightly.

The therapy was developed with the nonprofit n-Lorem Foundation, which screened more than 320 drug candidates before picking a lead. The findings were published in the journal Med.

The treatment remains experimental and is not standard care. The CHCHD10 mutation affects fewer than one per cent of people with hereditary ALS. A larger trial in eight more carriers is underway.