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

INTRODUCTION: Pendred syndrome (PDS)/DFNB 4 is a disorder with fluctuating and progressive hearing loss, vertigo, and thyroid goiter. We identified pathophysiology of a neurodegenerative disorder in PDS patient derived cochlear cells that were induced via induced pluripotent stem cells and found sir...

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Detalles Bibliográficos
Autores principales: Fujioka, Masato, Akiyama, Takumi, Hosoya, Makoto, Kikuchi, Kayoko, Fujiki, Yuto, Saito, Yasuko, Yoshihama, Keisuke, Ozawa, Hiroyuki, Tsukada, Keita, Nishio, Shin-ya, Usami, Shin-ichi, Matsunaga, Tatsuo, Hasegawa, Tomonobu, Sato, Yasunori, Ogawa, Kaoru
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Wolters Kluwer Health 2020
Materias:
6000
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7220392/
https://www.ncbi.nlm.nih.gov/pubmed/32384426
http://dx.doi.org/10.1097/MD.0000000000019763

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