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., …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
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