TY - RPRT TI - Dose-finding studies in drug development for rare genetic diseases AU - Wang, Lingshan AU - Wang, Jie AU - Feng, Ji AU - Doi, Mary AU - Pepe, Salvatore AU - Pacanowski, Michael AU - Schuck, Robert N. PY - 2022 DO - 10.1186/s13023-022-02298-6 UR - https://www.osti.gov/biblio/1983041 ID - 1983041 ER -