Onal presentations PubMed ID:https://www.ncbi.nlm.nih.gov/pubmed/24232037 at the reimbursement and licensing agencies. AFL, JJGMV, EWvZ, KJMS and SSW drafted the dossier ted for UNC1079 web scientific tips, with input from other authors. SSW and MCC analysed the scientific assistance on reimbursement and licensing. SSW drafted the manuscript, which was critically reviewed by all authors except YAH (deceased). All authors except YAH read and approved the final manuscript. Competing interests SSW, MRK, CV, KJMS, MCC, AFL, EWvZ and CGde W declare that they’ve no competing interests. JJGMV is a consultant for ArgenX and Alexion and participates inside a patent for MuSK myasthenia remedy. He has received grants from Duchenne Parent Project and Association contre les Myopathies Francaise, outside of your ted function; institutional grants from ZonMw, AFM France, Prinses Beatrix Spierfonds, and Spieren voor Spieren, outdoors from the ted perform. Consent for publication All sufferers offered written informed consent. Ethics approval and consent to participate Studying the trialists’ and patients’ perspective was specified inside the protocol registered under EudraCT quantity . The study protocol was approved by the Medical Ethics Committee from the Leiden University get RIP2 kinase inhibitor 1 Healthcare Center (reference no. P.), and all sufferers provided written informed consent. Assessment from the trial’s utility with respect to reimbursement and licensing was not ethically reviewed. The views expressed in this write-up will be the private views in the authors and may not be understood nor quoted as being made on behalf of, or reflecting the position of, the EMA or a single of its committees, operating parties or any in the national agencies.University Medical Center, Leiden, The Netherlands. Division of Health-related Statistics and Bioinformatics, Leiden University Health-related Center, Leiden, The Netherlands. Medicines Evaluation Board, Utrecht, The Netherlands. ReceivedFebruary AcceptedAprilPublisher’s NoteSpringer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. Author details Division of Clinical Genetics, Amsterdam Public Overall health research institute, VU University Healthcare Center, Amsterdam, The Netherlands. Division of Care, National Overall health Care Institute, Diemen, The Netherlands. Department of Epidemiology and Public Overall health, University College London, London, UK. Department of Neurology, Leiden University Health-related Center, Leiden, The Netherlands. Department of Clinical Pharmacy and Toxicology, LeidenReferences . Parliamentary documentation; Tweede Kamer der Staten Generaal. Visie op geneesmiddelenNieuwe geneesmiddelen snel bij de pati t tegen aanvaardbare kosten. Short van de minister van Volksgezondheid, Welzijn en Sport. View on medicinesnew medicines swiftly to individuals at acceptable costs. Letter in the Minister of Well being, Welfare and Sport. No January Available fromhttps:www.rijksoverheid. nldocumentenkamerstukkenkamerbriefovervi
sieopgeneesmiddelen. Accessed Jan National Overall health Care Institute (Zorginstituut Nederland). Farmacotherapeutisch Kompas. Vergoeding nietgeregistreerde indicaties, nietgeregistreerde geneesmiddelen en apotheekbereidingen Reimbursement of unlicensed indications, unlicensed medicines and compounded medicines . Accessed Apr . your next manuscript to BioMed Central and we’ll help you at every step:We accept presubmission inquiries Our selector tool aids you to seek out probably the most relevant journal We supply round the clock client support Practical on-line submission Thorough peer rev.Onal presentations PubMed ID:https://www.ncbi.nlm.nih.gov/pubmed/24232037 at the reimbursement and licensing agencies. AFL, JJGMV, EWvZ, KJMS and SSW drafted the dossier ted for scientific guidance, with input from other authors. SSW and MCC analysed the scientific advice on reimbursement and licensing. SSW drafted the manuscript, which was critically reviewed by all authors except YAH (deceased). All authors except YAH study and approved the final manuscript. Competing interests SSW, MRK, CV, KJMS, MCC, AFL, EWvZ and CGde W declare that they’ve no competing interests. JJGMV is really a consultant for ArgenX and Alexion and participates within a patent for MuSK myasthenia treatment. He has received grants from Duchenne Parent Project and Association contre les Myopathies Francaise, outside from the ted work; institutional grants from ZonMw, AFM France, Prinses Beatrix Spierfonds, and Spieren voor Spieren, outdoors from the ted operate. Consent for publication All individuals supplied written informed consent. Ethics approval and consent to participate Studying the trialists’ and patients’ perspective was specified within the protocol registered below EudraCT number . The study protocol was approved by the Health-related Ethics Committee in the Leiden University Healthcare Center (reference no. P.), and all patients offered written informed consent. Assessment in the trial’s utility with respect to reimbursement and licensing was not ethically reviewed. The views expressed within this article are the private views from the authors and may not be understood nor quoted as being produced on behalf of, or reflecting the position of, the EMA or 1 of its committees, working parties or any from the national agencies.University Healthcare Center, Leiden, The Netherlands. Department of Health-related Statistics and Bioinformatics, Leiden University Healthcare Center, Leiden, The Netherlands. Medicines Evaluation Board, Utrecht, The Netherlands. ReceivedFebruary AcceptedAprilPublisher’s NoteSpringer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. Author facts Division of Clinical Genetics, Amsterdam Public Overall health research institute, VU University Medical Center, Amsterdam, The Netherlands. Department of Care, National Overall health Care Institute, Diemen, The Netherlands. Division of Epidemiology and Public Health, University College London, London, UK. Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands. Division of Clinical Pharmacy and Toxicology, LeidenReferences . Parliamentary documentation; Tweede Kamer der Staten Generaal. Visie op geneesmiddelenNieuwe geneesmiddelen snel bij de pati t tegen aanvaardbare kosten. Brief van de minister van Volksgezondheid, Welzijn en Sport. View on medicinesnew medicines swiftly to sufferers at acceptable costs. Letter from the Minister of Overall health, Welfare and Sport. No January Obtainable fromhttps:www.rijksoverheid. nldocumentenkamerstukkenkamerbriefovervi
sieopgeneesmiddelen. Accessed Jan National Wellness Care Institute (Zorginstituut Nederland). Farmacotherapeutisch Kompas. Vergoeding nietgeregistreerde indicaties, nietgeregistreerde geneesmiddelen en apotheekbereidingen Reimbursement of unlicensed indications, unlicensed medicines and compounded medicines . Accessed Apr . your subsequent manuscript to BioMed Central and we’ll make it easier to at each step:We accept presubmission inquiries Our selector tool helps you to locate by far the most relevant journal We offer round the clock customer assistance Hassle-free on the web submission Thorough peer rev.