Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study

Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study

Abstract Background Facioscapulohumeral muscular dystrophy (FSHD) is a dominantly-inherited progressive muscular dystrophy caused by de-repression of the DUX4 gene, which causes disease by a toxic-gain-of-function. As molecularly targeted drugs move from preclinical testing into human trials, it is...

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Bibliographic Details
Main Authors: Samantha LoRusso, Nicholas E. Johnson, Michael P. McDermott, Katy Eichinger, Russell J. Butterfield, Elena Carraro, Kiley Higgs, Leann Lewis, Karlien Mul, Sabrina Sacconi, Valeria A. Sansone, Perry Shieh, Baziel van Engelen, Kathryn Wagner, Leo Wang, Jeffrey M. Statland, Rabi Tawil, on behalf of the ReSolve Investigators and the FSHD CTRN18
Format: Article
Language:English
Published: BMC 2019-09-01
Series:BMC Neurology
Subjects:
Facioscapulohumeral muscular dystrophy
Muscular dystrophy
Outcome measures
Clinical trial
Functional testing
Electrical impedance Myography
Online Access:http://link.springer.com/article/10.1186/s12883-019-1452-x

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http://link.springer.com/article/10.1186/s12883-019-1452-x

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