Funding for this research was provided by:
National Institute of Neurological Disorders and Stroke (U01NS101944)
Muscular Dystrophy Association (573783)
FSH Society (52016)
National Center for Advancing Translational Sciences (UL1TR000001, UL1RR024160)
French Muscular Dystrophy Association
Received: 30 April 2019
Accepted: 27 August 2019
First Online: 10 September 2019
Ethics approval and consent to participate
: The ReSolve study was reviewed by the University of Kansas Medical Center Central IRB using the SMART IRB reliance model, and all US sites agreed to rely on KUMC (KU IRB# STUDY00140842). Each site performed a local context review to adjust the informed consent to address any local considerations. European sites had a common protocol reviewed by their separate institution human subject committees. For the study site in Milan, Italy, the ethics review committee Comitato Etico Milano Area 3 reviewed and approved the study (reference number 88–032018). For the study site in Nijmegen, Netherlands, the ethics review committee CMO Arnhem-Nijmegen Region reviewed and approved the study (reference number NL64221.091.18). For the study site in Nice, France, the ethics review committee Comité de Protection des Personnes (C.P.P.) reviewed and approved the study (reference number 2019-A00658–49). All informed consent obtained from study participants is written.
: Not applicable
: The Reachable Workspace portion of this study was funded by Fulcrum Therapeutics.SL has no competing interests.NJ serves on scientific advisory boards for Cytokinetics, AveXis, AMO Pharma, and Biogen Idec; has received funding for travel and/or speaker honoraria from Strongbridge; serves as a consultant for AMO Pharma, AveXis, and Vertex Pharma; and receives research support from Ionis Pharmaceuticals, Biogen Idec, Valerion Therapeutics, Cytokinetics, Acceleron, AveXis, AMO Pharma, NIH/NINDS, FDA, Muscular Dystrophy Association, and Myotonic Dystrophy Foundation.MPM has no competing interests.KE has received consulting fees from Acceleron.RJB is supported by NIH grant 1K08NS097631–01. He is receiving funding via contracts for clinical trials from PTC Therapeutics, Sarepta Therapeutics, Pfizer, and Biogen. RJB serves on scientific advisory boards for Sarepta Therapeutics and Biogen.EC has no competing interests.KH has no competing interests.LL has no competing interests.KM has no competing interests.SS served as a consultant for Biomarin, Sanofi Genzyme, Alnylam Pharmaceuticals, Spark Therapeutic, Biotechspert and BIOGEN France.VAS serves on Scientific Advisory Boards for Biogen, PTC, Santhera, Sarepta, Avexis. VS has received research support from the Italian Telethon and Muscular Dystrophy Association grants.PS serves as a consultant for Avexis, Sarepta, and Biogen and has received speaker honoraria from Biogen, Alexion, Grifols, and CSL Behring.BvE reports grants from The Marigold Foundation and personal fees and non-financial support from Fulcrum and Facio, grants from Global FSH, Stichting Spieren voor Spieren, Prinses Beatrix Spierfonds, Dutch FSHD Foundation, European Union’s Horizon 2020 research and innovation programme (Murab), Netherlands Organisation for Scientific Research (NWO), The Netherlands Organisation for Health Research and Development (ZonMw), Association Francaise contre les Myopathies.KW served as a consultant for Sarepta, Pfizer, Wave, Fibrogen, and PTC Therapeutics.LW has no competing interests.JMS reports consulting work or serving on advisory boards for Fulcrum, Acceleron, Expansion, Biogen, Avexis, and Strongbridge.RT reports consulting work for Acceleron and serving on the Fulcrum Therapeutics advisory board.