Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool

Recent translational research developments in Spinal Muscular Atrophy (SMA), outcome measure design and demands from regulatory authorities require that clinical outcome assessments are ‘fit for purpose’. An international collaboration (SMA REACH UK, Italian SMA Network and PNCRN USA) undertook an i...

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Bibliografiske detaljer
Udgivet i:PLoS One
Main Authors: Ramsey, Danielle, Scoto, Mariacristina, Mayhew, Anna, Main, Marion, Mazzone, Elena S., Montes, Jacqueline, de Sanctis, Roberto, Dunaway Young, Sally, Salazar, Rachel, Glanzman, Allan M., Pasternak, Amy, Quigley, Janet, Mirek, Elizabeth, Duong, Tina, Gee, Richard, Civitello, Matthew, Tennekoon, Gihan, Pane, Marika, Pera, Maria Carmela, Bushby, Kate, Day, John, Darras, Basil T., De Vivo, Darryl, Finkel, Richard, Mercuri, Eugenio, Muntoni, Francesco
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2017
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5319655/
https://ncbi.nlm.nih.gov/pubmed/28222119
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0172346
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