1 Health Technology and Service Research, TechMed Centre, University of Twente, Enschede, Netherlands; Duchenne Parent Project, Veenendaal, Netherlands; International Rare Diseases Research Consortium, Paris, France.
2 International Rare Diseases Research Consortium, Paris, France; Eurordis-Rare Diseases Europe, Paris, France.
3 International Rare Diseases Research Consortium, Paris, France; ASrid, Tokyo, Japan.
4 International Rare Diseases Research Consortium, Paris, France; Sanford Research, Sioux Falls, SD 57104, USA; Sanford Health, Sioux Falls, SD, USA; Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD, USA. Electronic address: david.pearce@sanfordhealth.org.