Livrables
Motor emotional and learning deficits shared or unique to the different mouse models
First study approvals packageFirst study subject approvals package (prior to enrolment of first study subject). (i) Final version of study protocol as submitted to regulators/ethics committee(s), (ii) Registration number of clinical study in a WHO-or ICMJE- approved registry (with the possibility to post results), (iii) Approvals (ethics committees and national competent authority if applicable) required for invitation/enrolment of first subject in at least one clinical centre.
Communication, Dissemination and Exploitation plan (first release)First release of the Communication, Dissemination and Exploitation Plan.
First study subject approvals packageFirst study subject approvals package' (prior to enrolment of first study subject). (i) Final version of study protocol as submitted to regulators/ethics committee(s), (ii) Registration number of clinical study in a WHO-or ICMJE- approved registry (with the possibility to post results), (iii) Approvals (ethics committees and national competent authority if applicable) required for invitation/enrolment of first subject in at least one clinical centre.
Questionnaire definitionDefinition of a questionnaire that will be distributed via UPPMD to national advocacy groups and registries
Mdx5cv validationValidation of the mdx5cv mouse model
Gene and proteomic knowledge mapKnowledge map of gene and proteomic changes associated with comorbidities in other than DMDBMD
Comorbidity ‘supercluster' formationFormation of brain comorbidity supercluster and clinical trial advisory group on brain comorbidities
Brain transcriptional changesCharacterization of transcriptional changes in different brain regions of mouse models lacking different dystrophin isoforms
WP5 - Midterm recruitment reportMidterm recruitment report Report for each clinical study to be scheduled for the point when 50 of the study population is expected to have been recruited
Dystrophin isoform localisation:humansCharacterisation of dystrophin isoform localisation in different brain regions from normal human brains
Protocol optimisationOptimization of the neurocognitive and neurobehavioural protocols to be used in the study
Communication, Dissemination and Exploitation plan (second release)Second release of the Communication, Dissemination and Exploitation Plan.
WP6 - Midterm recruitment reportMidterm recruitment report Report for each clinical study to be scheduled for the point when 50 of the study population is expected to have been recruited
The project website will be formally delivered in month 4, carrying general information about BIND and the planned public deliverables; it will then be updated at regular intervals, to report on activities, events, major achievements and to provide access to public deliverables.
Publications
Auteurs:
A. Saoudi, S. Barberat, O. Le Coz, O. Vacca, M. Doisy Caquant, Tensorer, E. Sliwinski, L. Garcia, F. Muntoni, C. Vaillend and A. Goyenvalle
Publié dans:
Molecular Therapy Nucelic Acids, 2023, ISSN 2162-2531
Éditeur:
Nature Publishing Group
DOI:
10.1016/j.omtn.2023.03.009
Auteurs:
F. Zarrouki, S. Goutal, O. Vacca, L. Garcia, N. Tournier, A. Goyenvalle, and C. Vaillend
Publié dans:
International Journal of Molecular Sciences, 2022, ISSN 1422-0067
Éditeur:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/ijms232012617
Auteurs:
Francesco Muntoni and Mary Chessyre (UCL)
Publié dans:
Journal of Cachexia, Sarcopenia and Muscle, 2022, ISSN 2190-6009
Éditeur:
Wiley
DOI:
10.1002/jcsm.12914
Auteurs:
Cyrille Vaillend, A. Saoudi, F. Zarrouki, C. Sebrié, C. Izabelle, A. Goyenvalle, C. Vaillend
Publié dans:
Disease Models and Mechanisms, 2021, ISSN 1754-8411
Éditeur:
The company of Biologists
DOI:
10.1242/dmm.049028
Auteurs:
P. Weerkamp, E. Mol, D. Sweere, D. Schrans, R. J. Vermeulen, S. Klinkenberg, P. Hurks and J. Hendriksen
Publié dans:
Brain Sciences, 2022, ISSN 2076-3425
Éditeur:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/brainsci12111544
Auteurs:
P. Weerkamp, P. Collin, R. Maas, R. J. Vermeulen, S. Klinkenberg, J. Hendriksen
Publié dans:
Neuromuscular Disorders, 2022, ISSN 1873-2364
Éditeur:
Elsevier
DOI:
10.1016/j.nmd.2021.10.008
Auteurs:
A. Saoudi, C. Fergu, T. Gileadi, F. Montanaro, JE Morgan, V. Kelly, T. Tensorer, L. Garcia, C. Vaillend, F. Muntoni and A. Goyenvalle
Publié dans:
Cells, 2023, ISSN 2073-4409
Éditeur:
MDPI
DOI:
10.3390/cells12060908
Auteurs:
Faouzi Zarrouki
Karima Relizani Flavien Bizot
Thomas Tensorer
Luis Garcia
Cyrille Vaillend
Aurélie Goyenvalle
Publié dans:
Ann Neurol., 2022, ISSN 0364-5134
Éditeur:
John Wiley & Sons Inc.
DOI:
10.1002/ana.26409
Auteurs:
Hashimoto Y, Kuniishi H, Sakai K, Fukushima Y, Du X, Yamashiro K, Hori K, Motohashi N, Imamura M, Hoshino M, Yamada M, Araki T, Sakagami H, Takeda S, Itaka K, Ichinohe N, Muntoni F, Sekiguchi M, Aoki Y.
Publié dans:
Prog Neurobiol., 2021, ISSN 0301-0082
Éditeur:
Pergamon Press Ltd.
DOI:
10.1016/j.pneurobio.2022.102288
Auteurs:
Kate Maresh, Andriani Papageorgiou, Deborah Ridout, Neil A Harrison, William Mandy, David Skuse, Francesco Muntoni
Publié dans:
Research Article (peer reviewed), 2022, ISSN 1460-2156
Éditeur:
Oxford University Press
DOI:
10.1093/brain/awac048
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