@misc{indiciaea3642ebdbb43, title = {Dose-finding studies in drug development for rare genetic diseases}, author = {Wang, Lingshan and Wang, Jie and Feng, Ji and Doi, Mary and Pepe, Salvatore and Pacanowski, Michael and Schuck, Robert N.}, year = {2022}, doi = {10.1186/s13023-022-02298-6}, url = {https://www.osti.gov/biblio/1983041}, note = {Source identifier: 1983041} }