#GeneTherapy

The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical milestone.

Researchers say a gene therapy allowed deaf children and adults as old as 32 to hear for the first time. The benefits have persisted for more than two years for some patients.

For the second time, Vinay Prasad is set to leave the Food and Drug Administration.

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.