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

Abstract:

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 benefit, and comprehensive information about the natural history of the disease are essential for regulatory approval of any treatment. However, for Duchenne muscular dystrophy and other rare diseases...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Accepted Manuscript

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Publisher copy:
10.1016/S1474-4422(16)30035-7

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Publisher:
Elsevier Publisher's website
Journal:
Lancet Neurology Journal website
Volume:
15
Issue:
8
Pages:
882-890
Publication date:
2016-07-05
DOI:
EISSN:
1474-4465
ISSN:
1474-4422
Pubs id:
pubs:652027
URN:
uri:022629c9-5c9f-4039-b89f-4b3153c5efee
UUID:
uuid:022629c9-5c9f-4039-b89f-4b3153c5efee
Local pid:
pubs:652027
Paper number:
8
Language:
English
Keywords:

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