One in a Million: Speedy Work from Maine to California Leads to Custom Genetic Treatment for Rare Disease

The therapy and its approach generate hope for afflicted families

KJ Muldoon after the genetic therapy

The arguably biggest biomedical story of the year popped up in Philadelphia last week and involved a nine-month-old baby, a one-in-a-million rare disease and a 100-person team scattered across the country, including California.

The best summary of the tale might be the headlines on the Wired website:

“A Baby Received a …