An experimental therapy saves the lives of more than 60 children with a deadly disease

An experimental gene therapy has saved the lives of 62 children affected by the extremely cruel ADA-SCID, a genetic childhood disease that weakens the immune system and facilitates all kinds of deadly infections: pneumonia, meningitis, chickenpox. The father of one of these children, Jeff Nachem, detailed over the phone the ordeal his family endured a decade ago. “It was very hard. Our daughter Eliana had to be confined to the house, so my wife also isolated herself from the world so as not to risk infecting her. I had to keep going to work, but when I returned home I had to shower and put on clean clothes before touching them,” he recalls from New York. The revolutionary treatment is administered in just one single dose, with no need for further doses, but it is so sophisticated that it can cost around one million euros. Eliana received it a decade ago and now leads a completely normal life. “She gets great grades in school, plays basketball, and has even joined the school choir. It’s incredible,” her father recounts with emotion.

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