Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

Duchenne muscular dystrophy is a rare, progressive, muscle-wasting disease leading to severe disability and premature death. Treatment is currently symptomatic, but several experimental therapies are in development. Implemented care standards, validated outcome measures correlating with clinical ben...

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Bibliographic Details
Main Authors: Straub, V, Balabanov, P, Bushby, K, Ensini, M, Goemans, N, De Luca, A, Pereda, A, Hemmings, R, Campion, G, Kaye, E, Arechavala-Gomeza, V, Goyenvalle, A, Niks, E, Veldhuizen, O, Furlong, P, Stoyanova-Beninska, V, Wood, M, Johnson, A, Mercuri, E, Muntoni, F, Sepodes, B, Haas, M, Vroom, E, Aartsma-Rus, A
Format: Journal article
Language:English
Published: Elsevier 2016

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