A phase I/IIa double blind single institute trial of low dose sirolimus for Pendred syndrome/DFNB4.

Fujioka M, Akiyama T, Hosoya M, Kikuchi K, Fujiki Y, Saito Y, Yoshihama K, Ozawa H, Tsukada K, Nishio S-Y, et al. A phase I/IIa double blind single institute trial of low dose sirolimus for Pendred syndrome/DFNB4. Medicine (Baltimore). 2020;99(19):e19763.