First drug registry in Duchenne muscular dystrophy (DMD) to assess Translarna (Ataluren) use, safety, and effectiveness in routine clinical practice

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Author(s): Bushby K, Reha A, Northcutt V, Luo X, Ong T, Park S, Spiegel R, Transiarna Patient Registry

Publication type: Conference Proceedings (inc. Abstract)

Publication status: Published

Conference Name: 20th International Congress of the The World Muscle Society

Year of Conference: 2015

Pages: S259

Print publication date: 01/10/2015

Online publication date: 10/09/2015

Acceptance date: 01/01/1900

ISSN: 0960-8966

Publisher: Elsevier

URL: https://doi.org/10.1016/j.nmd.2015.06.268

DOI: 10.1016/j.nmd.2015.06.268

Series Title: Neuromuscular Disorders

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