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Facioscapulohumeral Muscular Dystrophy European Patient Survey: Assessing Patient Reported Disease Burden and Preferences in Clinical Trial Participation.

Megan M. McNiff, Sheila Hawkins, Bine Haase, Joanne Bullivant,Tammy McIver, Olga Mitelman,Nicholas Emery,Giorgio Tasca,Nicol Voermans,Jordi Diaz-Manera

JOURNAL OF NEUROMUSCULAR DISEASES(2024)

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关键词
Facioscapulohumeral muscular dystrophy (FSHD),patient preferences,patient reported outcomes,decision-making,clinical trials,therapies
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