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 SY, Usami SI, Matsunaga T, Hasegawa T, Sato Y, Ogawa K.
Fujioka M, et al.
Medicine (Baltimore). 2020 May;99(19):e19763. doi: 10.1097/MD.0000000000019763.
Medicine (Baltimore). 2020.
PMID: 32384426
Free PMC article.
Clinical Trial.
INTRODUCTION: Pendred syndrome (PDS)/DFNB 4 is a disorder with fluctuating and progressive hearing loss, vertigo, and thyroid goiter. ...TRIAL REGISTRATION NUMBER: JMA-IIA00361; Pre-results....
INTRODUCTION: Pendred syndrome (PDS)/DFNB 4 is a disorder with fluctuating and progressive hearing loss, vertigo, and thyroid …