Bespoke RNA Drug Reverses ALS

JacksonvilleA physician with ALS improved, not declined.

The disease normally only worsens, so any reversal reshapes what’s possible for rare-mutation cases.

The drug, built against his single CHCHD10 mutation, silences the faulty RNA transcript rather than editing his genes.

Researchers call it an early, one-patient result and plan two more years of monitoring.

Sources: Nature