The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development

Heslop, E; Csimma, C; Straub, V; McCall, J; Nagaraju, K; Wagner, K R; Caizergues, D; Korinthenberg, R; Flanigan, K M; Kaufmann, P; McNeil, E; Mendell, J; Hesterlee, S; Wells, D J

doi:10.1186/s13023-015-0258-1

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development

Heslop, E; Csimma, C; Straub, V; McCall, J; Nagaraju, K; Wagner, K R; Caizergues, D; Korinthenberg, R; Flanigan, K M; Kaufmann, P; McNeil, E; Mendell, J; Hesterlee, S; Wells, D J

Authors

E Heslop

C Csimma

V Straub

J McCall

K Nagaraju

K R Wagner

D Caizergues

R Korinthenberg

K M Flanigan

P Kaufmann

E McNeil

J Mendell

S Hesterlee

D J Wells

Citation

Heslop, E., Csimma, C., Straub, V., McCall, J., Nagaraju, K., Wagner, K. R., Caizergues, D., Korinthenberg, R., Flanigan, K. M., Kaufmann, P., McNeil, E., Mendell, J., Hesterlee, S., & Wells, D. J. (in press). The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. Orphanet Journal of Rare Diseases, 10(49), https://doi.org/10.1186/s13023-015-0258-1

Journal Article Type	Article
Acceptance Date	Mar 25, 2015
Deposit Date	May 22, 2015
Publicly Available Date	May 22, 2015
Journal	ORPHANET JOURNAL OF RARE DISEASES
Electronic ISSN	1750-1172
Publisher	BioMed Central
Peer Reviewed	Peer Reviewed
Volume	10
Issue	49
DOI	https://doi.org/10.1186/s13023-015-0258-1
Public URL	https://rvc-repository.worktribe.com/output/1401492