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

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 essentia...

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Detalles Bibliográficos
Autores principales: LoRusso, Samantha, Johnson, Nicholas E., McDermott, Michael P., Eichinger, Katy, Butterfield, Russell J., Carraro, Elena, Higgs, Kiley, Lewis, Leann, Mul, Karlien, Sacconi, Sabrina, Sansone, Valeria A., Shieh, Perry, van Engelen, Baziel, Wagner, Kathryn, Wang, Leo, Statland, Jeffrey M., Tawil, Rabi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Study Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6734593/
https://www.ncbi.nlm.nih.gov/pubmed/31506080
http://dx.doi.org/10.1186/s12883-019-1452-x

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