Resultado final
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.
Publicaciones
Autores:
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.
Publicado en:
2021
Editor:
Elsevier
DOI:
10.1016/j.pneurobio.2022.102288
Autores:
Kate Maresh, Andriani Papageorgiou, Deborah Ridout, Neil A Harrison, William Mandy, David Skuse, Francesco Muntoni
Publicado en:
Research Article (peer reviewed), 2022, ISSN 1460-2156
Editor:
Oxford University Press
DOI:
10.1093/brain/awac048
Autores:
A. Saoudi, S. Barberat, O. Le Coz, O. Vacca, M. Doisy Caquant, Tensorer, E. Sliwinski, L. Garcia, F. Muntoni, C. Vaillend and A. Goyenvalle
Publicado en:
Molecular Therapy Nucelic Acids, 2023, ISSN 2162-2531
Editor:
Nature Publishing Group
DOI:
10.1016/j.omtn.2023.03.009
Autores:
F. Zarrouki, S. Goutal, O. Vacca, L. Garcia, N. Tournier, A. Goyenvalle, and C. Vaillend
Publicado en:
International Journal of Molecular Sciences, 2022, ISSN 1422-0067
Editor:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/ijms232012617
Autores:
Francesco Muntoni and Mary Chessyre (UCL)
Publicado en:
Journal of Cachexia, Sarcopenia and Muscle, 2022, ISSN 2190-6009
Editor:
Wiley
DOI:
10.1002/jcsm.12914
Autores:
Cyrille Vaillend, A. Saoudi, F. Zarrouki, C. Sebrié, C. Izabelle, A. Goyenvalle, C. Vaillend
Publicado en:
Disease Models and Mechanisms, 2021, ISSN 1754-8411
Editor:
The company of Biologists
DOI:
10.1242/dmm.049028
Autores:
P. Weerkamp, E. Mol, D. Sweere, D. Schrans, R. J. Vermeulen, S. Klinkenberg, P. Hurks and J. Hendriksen
Publicado en:
Brain Sciences, 2022, ISSN 2076-3425
Editor:
Multidisciplinary Digital Publishing Institute (MDPI)
DOI:
10.3390/brainsci12111544
Autores:
P. Weerkamp, P. Collin, R. Maas, R. J. Vermeulen, S. Klinkenberg, J. Hendriksen
Publicado en:
Neuromuscular Disorders, 2022, ISSN 1873-2364
Editor:
Elsevier
DOI:
10.1016/j.nmd.2021.10.008
Autores:
A. Saoudi, C. Fergu, T. Gileadi, F. Montanaro, JE Morgan, V. Kelly, T. Tensorer, L. Garcia, C. Vaillend, F. Muntoni and A. Goyenvalle
Publicado en:
Cells, 2023, ISSN 2073-4409
Editor:
MDPI
DOI:
10.3390/cells12060908
Autores:
Faouzi Zarrouki
Karima Relizani Flavien Bizot
Thomas Tensorer
Luis Garcia
Cyrille Vaillend
Aurélie Goyenvalle
Publicado en:
Ann Neurol., 2022, ISSN 0364-5134
Editor:
John Wiley & Sons Inc.
DOI:
10.1002/ana.26409
Autores:
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.
Publicado en:
Prog Neurobiol., 2021, ISSN 0301-0082
Editor:
Pergamon Press Ltd.
DOI:
10.1016/j.pneurobio.2022.102288
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