Miracle surgery saves man with rare brain bubble at NYU Langone

A child un­der two years old who re­ceived an ex­per­i­men­tal gene ther­a­py from Re­gen­eron in one ear saw im­proved au­di­to­ry re­spons­es through six weeks, ac­cord­ing

The FDA re­ject­ed Rock­et Phar­ma­ceu­ti­cals’ gene ther­a­py for leuko­cyte ad­he­sion de­fi­cien­cy-I (LAD-I), re­quest­ing “lim­it­ed ad­di­tion­al” man­u­fac­tur­ing in­for­ma­tion. The com­pa­ny an­nounced the … Sign up to