Deliverables Documents, reports (14) Mdx intellectual defects Motor emotional and learning deficits shared or unique to the different mouse models First study approvals package First 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 package First 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 definition Definition of a questionnaire that will be distributed via UPPMD to national advocacy groups and registries Mdx5cv validation Validation of the mdx5cv mouse model Gene and proteomic knowledge map Knowledge map of gene and proteomic changes associated with comorbidities in other than DMDBMD Comorbidity ‘supercluster' formation Formation of brain comorbidity supercluster and clinical trial advisory group on brain comorbidities Brain transcriptional changes Characterization of transcriptional changes in different brain regions of mouse models lacking different dystrophin isoforms WP5 - Midterm recruitment report Midterm 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:humans Characterisation of dystrophin isoform localisation in different brain regions from normal human brains Protocol optimisation Optimization 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 report Midterm 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 Websites, patent fillings, videos etc. (1) Project Website 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 Conference proceedings (2) Brain Dp140 alters glutamatergic transmission and social behaviour in the mdx52 model of Duchenne muscular dystrophy Author(s): 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. Published in: 2021 Publisher: Elsevier DOI: 10.1016/j.pneurobio.2022.102288 Startle responses in Duchenne muscular dystrophy: a novel biomarker of brain dystrophin deficiency Author(s): Kate Maresh, Andriani Papageorgiou, Deborah Ridout, Neil A Harrison, William Mandy, David Skuse, Francesco Muntoni Published in: Research Article (peer reviewed), 2022, ISSN 1460-2156 Publisher: Oxford University Press DOI: 10.1093/brain/awac048 Peer reviewed articles (9) Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotidesalleviates emotional deficits in mdx52 mice Author(s): A. Saoudi, S. Barberat, O. Le Coz, O. Vacca, M. Doisy Caquant, Tensorer, E. Sliwinski, L. Garcia, F. Muntoni, C. Vaillend and A. Goyenvalle Published in: Molecular Therapy Nucelic Acids, 2023, ISSN 2162-2531 Publisher: Nature Publishing Group DOI: 10.1016/j.omtn.2023.03.009 Abnormal Expression of Synaptic and Extrasynaptic GABAA Receptor Subunits in the Dystrophin-Deficient mdx Mouse Author(s): F. Zarrouki, S. Goutal, O. Vacca, L. Garcia, N. Tournier, A. Goyenvalle, and C. Vaillend Published in: International Journal of Molecular Sciences, 2022, ISSN 1422-0067 Publisher: Multidisciplinary Digital Publishing Institute (MDPI) DOI: 10.3390/ijms232012617 Duchenne muscular dystrophy patients lacking the dystrophin isoforms Dp140 and Dp71 and mouse models lacking Dp140 have a more severe motor phenotype Author(s): Francesco Muntoni and Mary Chessyre (UCL) Published in: Journal of Cachexia, Sarcopenia and Muscle, 2022, ISSN 2190-6009 Publisher: Wiley DOI: 10.1002/jcsm.12914 Emotional behavioral and brain anatomy of the mdx52 mouse model of Duchenne muscular dystrophy Author(s): Cyrille Vaillend, A. Saoudi, F. Zarrouki, C. Sebrié, C. Izabelle, A. Goyenvalle, C. Vaillend Published in: Disease Models and Mechanisms, 2021, ISSN 1754-8411 Publisher: The company of Biologists DOI: 10.1242/dmm.049028 Wechsler Scale Intelligence Testing in Males with Dystrophinopathies: A Review and Meta-Analysis Author(s): P. Weerkamp, E. Mol, D. Sweere, D. Schrans, R. J. Vermeulen, S. Klinkenberg, P. Hurks and J. Hendriksen Published in: Brain Sciences, 2022, ISSN 2076-3425 Publisher: Multidisciplinary Digital Publishing Institute (MDPI) DOI: 10.3390/brainsci12111544 Psychosocial adjustment in adults with Duchenne muscular dystrophy: A pilot study on a shortened parent-report questionnaire Author(s): P. Weerkamp, P. Collin, R. Maas, R. J. Vermeulen, S. Klinkenberg, J. Hendriksen Published in: Neuromuscular Disorders, 2022, ISSN 1873-2364 Publisher: Elsevier DOI: 10.1016/j.nmd.2021.10.008 Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models Author(s): A. Saoudi, C. Fergu, T. Gileadi, F. Montanaro, JE Morgan, V. Kelly, T. Tensorer, L. Garcia, C. Vaillend, F. Muntoni and A. Goyenvalle Published in: Cells, 2023, ISSN 2073-4409 Publisher: MDPI DOI: 10.3390/cells12060908 Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X-Linked (mdx) Mouse Author(s): Faouzi Zarrouki Karima Relizani Flavien Bizot Thomas Tensorer Luis Garcia Cyrille Vaillend Aurélie Goyenvalle Published in: Ann Neurol., 2022, ISSN 0364-5134 Publisher: John Wiley & Sons Inc. DOI: 10.1002/ana.26409 Brain Dp140 alters glutamatergic transmission and social behaviour in the mdx52 model of Duchenne muscular dystrophy Author(s): 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. Published in: Prog Neurobiol., 2021, ISSN 0301-0082 Publisher: Pergamon Press Ltd. DOI: 10.1016/j.pneurobio.2022.102288 Searching for OpenAIRE data... 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