Quantitative retrospective natural history modeling for orphan drug development.
Garbade SF, Zielonka M, Komatsuzaki S, Kölker S, Hoffmann GF, Hinderhofer K, Mountford WK, Mengel E, Sláma T, Mechler K, Ries M.
Garbade SF, et al. Among authors: kolker s.
J Inherit Metab Dis. 2021 Jan;44(1):99-109. doi: 10.1002/jimd.12304. Epub 2020 Sep 8.
J Inherit Metab Dis. 2021.
PMID: 32845020
Review.