Suchergebnisse

Altres ajuts: C. E. Teunissen PhD received grants from the European Commission, the Dutch Research Council (ZonMW), Association of Frontotemporal Dementia/Alzheimer's Drug Discovery Foundation, Alzheimer Netherlands. M. Otto MD PhD received funding from the EU, German ministry of science and technology (KNDD/FTLD) the Baden-Wuertemberg Foundation. Sebastiaan Engelborghs MD, PhD received funding from the EU/EFPIA Innovative Medicines Initiative Joint Undertaking (EMIF grant number 115372); the University Research Fund of the University of Antwerp; the Flemish Impulse Financing of Networks for Dementia Research (VIND); and Flanders Innovation & Entrepreneurship (VLAIO). Sanna-Kaisa Herukka MD, PhD received funding from Finnish Medical Foundation and Finnish Medical Society Duodecim. Sylvain Lehmann MD, PhD has received support from "France Alzheimer", the National French Alzheimer effort ("Plan Alzheimer"). Piotr Lewczuk MD, PhD has received support from the Innovative Medicines Initiative Joint Undertaking under EMIF grant agreement number 115372, resources of which are composed of financia contributions from the European Union's Seventh Framework Programme (FP7/2007-2013) and EFPIA companies' in kind contribution. Hayrettin Tumani MD received research support from Hertie-Stiftung, BMBF, University of Ulm and Landesstiftung BW. Martin R. Turner MBBS, PhD is supported by the Medical Research Council & Motor Neurone Disease Association Lady Edith Wolfson Senior Clinical Fellowship (MR/K01014X/1). MM Verbeek PhD received grants from Alzheimer Nederland (number 14502) and the CAVIA project, which has been made possible by ZonMW (number 733050202). The CAVIA project is part of 'Memorabel', the research and innovation programme for dementia, as part of the Dutch national 'Deltaplan for Dementia': zonmw.nl/dementiaresearch. The CAVIA project is a consortium of Radboudumc, LUMC, Erasmus MC, VUmc, ADX Neurosciences, Philips Healthcare, Stony Brook University and Massachusetts General Hospital. Jens Wiltfang is ...

Background: Spinocerebellar ataxia type 3 is a rare neurodegenerative disease caused by a CAG repeat expansion in the ataxin-3 gene. Although no curative therapy is yet available, preclinical gene-silencing approaches to reduce polyglutamine (polyQ) toxicity demonstrate promising results. In view of upcoming clinical trials, quantitative and easily accessible molecular markers are of critical importance as pharmacodynamic and particularly as target engagement markers. Objective: We aimed at developing an ultrasensitive immunoassay to measure specifically polyQ-expanded ataxin-3 in plasma and cerebrospinal fluid (CSF). Methods: Using the novel single molecule counting ataxin-3 immunoassay, we analyzed cross-sectional and longitudinal patient biomaterials. Results: Statistical analyses revealed a correlation with clinical parameters and a stability of polyQ-expanded ataxin-3 during conversion from the pre-ataxic to the ataxic phases. Conclusions: The novel immunoassay is able to quantify polyQ-expanded ataxin-3 in plasma and CSF, whereas ataxin-3 levels in plasma correlate with disease severity. Longitudinal analyses demonstrated a high stability of polyQ-expanded ataxin-3 over a short period. © 2021 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society ; Funding agencies: This project is supported by the EU Joint Programme—Neurodegenerative Disease Research (JPND) through the following funding organizations under the aegis of JPND: Germany, Federal Ministry of Education and Research (BMBF; funding codes 01ED1602A/B); Netherlands, The Netherlands Organisation for Health Research and Development; Portugal, Foundation for Science and Technology (FCT, grant number JPCOFUND/0001/2015), and Regional Fund for Science and Technology of the Azores; and United Kingdom, Medical Research Council. This project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement number 643417. In addition, support has been received by the BIONIC project (number 733050822, which has been made possible by ZonMW as part of "Memorabel," the research and innovation program for dementia, as part of the Dutch national "Deltaplan for Dementia": zonmw.nl/dementiaresearch), the CAF[1]E project (the National Institutes of Health, USA, grant number 5R01NS104147-02), and a grant from the Selfridges Group Foundation (NR170024). The BIONIC project is a consortium of Radboudumc, LUMC, ADX Neurosciences, and Rhode Island University.