Back to matchesWe found a matchYour institution may have rights to this item. Sign in to continue.TitleQuantitative retrospective natural history modeling for orphan drug development.AuthorsGarbade, Sven F.; Zielonka, Matthias; Komatsuzaki, Shoko; Kölker, Stefan; Hoffmann, Georg F.; Hinderhofer, Katrin; Mountford, William K.; Mengel, Eugen; Sláma, Tomáš; Mechler, Konstantin; Ries, MarkusPublicationJournal of Inherited Metabolic Disease, 2021, Vol 44, Issue 1, p99ISSN0141-8955Publication typeArticleDOI10.1002/jimd.12304