Stakeholder collaboration for spinal muscular atrophy therapy development

Several topics discussed at the stakeholder meeting were the same as those identified in the Duchenne muscular dystrophy discussion, including variability of standards of care; need for developing outcome measures based on what patients find important; collecting natural history; and identifying and...

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Bibliographic Details
Published in:Lancet neurology 2017-04, Vol.16 (4), p.264-264
Main Authors: Aartsma-Rus, Annemieke, Balabanov, Pavel, Binetti, Luca, Haas, Manuel, Haberkamp, Marion, Mitchell, Joanna, Rosa, Mário Miguel, Muntoni, Francesco, Finkel, Richard, Mercuri, Eugenio
Format: Article
Language:English
Subjects:
Collaboration
Humans
Muscular Atrophy, Spinal
Muscular dystrophy
Neurology
Spinal Muscular Atrophies of Childhood
Stakeholders
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Summary:Several topics discussed at the stakeholder meeting were the same as those identified in the Duchenne muscular dystrophy discussion, including variability of standards of care; need for developing outcome measures based on what patients find important; collecting natural history; and identifying and validating appropriate tools for efficacy assessment, reflecting objective and clinically significant outcomes.1 Other topics were more specific to SMA, including the choice of appropriate controls in clinical trials, the huge burden for families involved in around the clock care for their child, and how trial participation contributes to this burden.
ISSN:1474-4422
1474-4465
DOI:10.1016/S1474-4422(17)30041-8