One treatment, a corrected gene: early results for children born with a rare sugar disorder

Babies born with glycogen storage disease type Ia cannot make their own glucose properly, so families live by the clock, feeding around the clock to keep blood sugar safe. Beam Therapeutics reports its one-time base-editing treatment restored the body's own glucose production in an early trial, with improvements growing at higher doses. The full data will be presented at a gene-therapy congress in Hamburg this month.