Framing childhood-onset facioscapulohumeral dystrophy: from first symptoms to future trials

Jildou N. Dijkstra has nothing to report. Corrie E. Erasmus has the following declarations of interest: co-chair Working group Pediatrics of the European Trial Network FSHD, board member Duchenne Center Netherlands, advisory board Pfizer, Roche. Research grants received from the Dutch Prinses Beatrix Spier-fonds, Spieren voor Spieren, FSHD Stichting. Pierre Laurian has nothing to report. Ally Roets has the following declarations of interest: Director, Parent Chapter, FSHD Society and Vice President, Cure FSHD Initiative. Matthew Dodd has nothing to report. Cristiane Moreno has nothing to report. Ian Woodcock has the following declarations of interest: received honoraria for work performed including educational activities and attendance at advisory board meetings from pharmaceutical companies Avidity, Biogen, Juvena Therapeutics, Keros Therapeutics, Novartis, Pfizer, Roche, Solid Biosciences and Souffle Therapeutics. Dr Woodcock has received grants for research work from Fulcrum Therapeutics, FSHD Global Research Foundation, FSHD Society, NIH. Dr Woodcock has been principal investigator on a number of industry-sponsored clinical trials, including those sponsored by pharmaceutical companies Avidity, Bio-gen, Catabasis, Dyne Therapeutics, EryDel, Keros Therapeutics, Novar-tis, Pfizer, Percheron Therapeutics, PTC, Roche, Sarepta, Satellos, and Solid Biosciences. None of these disclosures affected the work Dr Woodcock performed on this work. Kathryn Irving has nothing to report. Nicol C. Voermans has the following declarations of interest: leader of the FSHD European Trial Network. Honoraria for consultancy or advisory board participation from: Fulcrum, Dyne, Avidity, Souffle, Argenx, and Astellas (all paid to the hospital) . Tracey Willis has the following declarations of interest: co-chair