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

Lookup NU author(s): Emma Heslop, Professor Volker Straub, Emerita Professor Katherine Bushby



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).

Publication metadata

Author(s): Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K

Publication type: Article

Publication status: Published

Journal: Orphanet Journal of Rare Diseases

Year: 2015

Volume: 10

Online publication date: 23/04/2015

Acceptance date: 25/03/2015

Date deposited: 27/05/2015

ISSN (electronic): 1750-1172

Publisher: BioMed Central Ltd


DOI: 10.1186/s13023-015-0258-1


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