Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a larg...
Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study
About this item
Full title
Author / Creator
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 , ReSolve Investigators and the FSHD CTRN18 and on behalf of the ReSolve Investigators and the FSHD CTRN18
Publisher
England: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: BioMed Central Ltd
Subjects
More information
Scope and Contents
Contents
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 essential that we validate clinical trial tools and methodology to fac...
Alternative Titles
Full title
Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study
Authors, Artists and Contributors
Author / Creator
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
ReSolve Investigators and the FSHD CTRN18
on behalf of the ReSolve Investigators and the FSHD CTRN18
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_9f896a44cef44c188a88370316d2d5e1
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_9f896a44cef44c188a88370316d2d5e1
Other Identifiers
ISSN
1471-2377
E-ISSN
1471-2377
DOI
10.1186/s12883-019-1452-x
