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The risks of therapeutic misconception and individual patient (n = 1) “trials” in rare diseases such as Duchenne dystrophy

Lookup NU author(s): Emma Heslop, Dr Pauline McCormack, Professor Christoph Rehmann-Sutter, Emeritus Professor Simon WoodsORCiD

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Author(s): Aartsma-Rus A, Cosigned by, Furlong P, Vroom E, van Ommen GJ, Niks E, Straathof C, Verschuuren J, Ferlini A, Hagger L, Heslop E, Karcagi V, Kirschner J, McCormack P, Moeschen P, Muntoni F, Ouillade MC, Rahbeck J, Rehmann-Sutter C, Rouault F, Sejersen T, Woods S

Publication type: Article

Publication status: Published

Journal: Neuromuscular Disorders

Year: 2011

Volume: 21

Issue: 1

Pages: 13-15

Print publication date: 03/11/2010

Date deposited: 07/01/2013

ISSN (print): 0960-8966

ISSN (electronic): 1873-2364

Publisher: Elsevier

URL: http://dx.doi.org/10.1016/j.nmd.2010.09.012

DOI: 10.1016/j.nmd.2010.09.012


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