Marshall, Randall D.
Collins, Abigail
Escolar, Maria L.
Jinnah, H. A.
Klopstock, Thomas
Kruer, Michael C.
Videnovic, Aleksandar
Robichaux-Viehoever, Amy
Burns, Colleen
Swett, Laura L.
Revicki, Dennis A.
Bender, Randall H.
Lenderking, William R.
Funding for this research was provided by:
Retrophin, Inc. (Not applicable)
Article History
Received: 1 March 2019
Accepted: 25 June 2019
First Online: 12 July 2019
Ethics approval and consent to participate
: The study protocol received prior approval by Ethical and Independent Review Services, a central IRB. Interested potential participants were mailed a study packet that included two copies of the informed consent and assent forms. Evidera staff explained the study by telephone and obtained written and verbal informed consent for participants prior to the first data collection interview. Participants returned the signed informed consent forms in a postage-paid envelope.
: Not applicable.
: RDM was formerly an employee of Retrophin, Inc. and may have an equity or other financial interest in Retrophin, Inc.AC has provided consultant services to Ultragenyx, Inc. and Retrophin, Inc.MLE has provided consultant services to Retrophin, Inc.HAJ has provided consultant services to Retrophin, Inc. and has active or recent grant support from the US government (National Institutes of Health), private philanthropic organizations (the Benign Essential Blepharospasm Research Foundation, Cure Dystonia Now), academically-oriented institutions (the Dystonia Study Group), and industry (Cavion Therapeutics, Ipsen Pharmaceuticals, Retrophin Inc.); has served on advisory boards or as a consultant for Abide Therapeutics, Allergan Inc., Psyadon Pharmaceuticals, Saol Therapeutics, and Medtronic Inc.; has received honoraria or stipends for lectures or administrative work from the American Academy of Neurology, the Dystonia Medical Research Foundation, the International Neurotoxin Society, the International Parkinson’s Disease and Movement Disorders Society, The Parkinson’s Disease Foundation, and Tyler’s Hope for a Cure; serves on the Scientific Advisory Boards for Cure Dystonia Now, the Dystonia Medical Research Foundation, Lesch-Nyhan Action France, and Tyler’s Hope for a Cure; and is principle investigator for the Dystonia Coalition, which receives the majority of its support through NIH grant TR001456 from the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences, and previously NS065701 from the National Institutes of Neurological Disorders and Stroke. The Dystonia Coalition has received additional material or administrative support from industry sponsors (Allergan Inc. and Merz Pharmaceuticals) as well as private foundations (The American Dystonia Society, Beat Dystonia, The Benign Essential Blepharospasm Foundation, Cure Dystonia Now, Dystonia Inc., Dystonia Ireland, The Dystonia Medical Research Foundation, The European Dystonia Federation, The Foundation for Dystonia Research, The National Spasmodic Dysphonia Association, and The National Spasmodic Torticollis Association).TK did not receive any payment or honoraria from Retrophin, Inc. when the research was conducted, but is currently the coordinating investigator of a clinical study of fosmetpantotenate in PKAN, which is sponsored by Retrophin, Inc. He acknowledges support by the European Commission 7th Framework Programme (FP7/2007–2013, HEALTH-F2–2011, grant agreement No. 277984, TIRCON) and by the European Reference Network for Rare Neurological Diseases (ERN-RND) co-funded by the European Commission (ERN-RND: 3HP 767231). He is a coordinating investigator of the deferiprone in PKAN randomized and extension trial and received research funding from ApoPharma Inc. He provides consulting services to CoA Therapeutics and TM3 Therapeutics. He has received travel support from ApoPharma Inc.MCK has received grant support from Retrophin, Inc. and receives funding from both federal sources (US NIH NS106298) and foundation sources (Scott Foundation, CureCP, Cerebral Palsy Alliance Research Foundation). He serves on the Scientific Advisory Board of the Spastic Paraplegia Foundation and has served as a speaker for the Tourette Association of America and as a consultant for PTC Therapeutics.AV has provided consultant services to Retrophin, Inc.ARV has received grant funding from NIH and the McDonnell Center for Systems Neuroscience.CB is an employee of Retrophin, Inc. and may have an equity or other financial interest in Retrophin, Inc.LLS, DAR, and WRL are employees of Evidera, and were hired by Retrophin, Inc. to conduct the research. RHB was an employee of Evidera when the research was conducted. They did not receive any payment or honoraria directly from Retrophin, Inc. for services rendered. Evidera received funding from Retrophin, Inc. to participate in the study and the development of the first draft of this manuscript.