Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
Tambuyzer E, Vandendriessche B, Austin CP, Brooks PJ, Larsson K, Miller Needleman KI, Valentine J, Davies K, Groft SC, Preti R, Oprea TI, Prunotto M.
Tambuyzer E, et al. Among authors: davies k.
Nat Rev Drug Discov. 2020 Feb;19(2):93-111. doi: 10.1038/s41573-019-0049-9. Epub 2019 Dec 13.
Nat Rev Drug Discov. 2020.
PMID: 31836861
Review.