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Recent Advances in Clinical Trials in Multiple System Atrophy

dc.rights.licenseopenen_US
hal.structure.identifierInstitut des Maladies Neurodégénératives [Bordeaux] [IMN]
dc.contributor.authorBENDETOWICZ, David
dc.contributor.authorFABBRI, Margherita
dc.contributor.authorSIRNA, Federico
dc.contributor.authorFERNAGUT, Pierre-Olivier
hal.structure.identifierInstitut des Maladies Neurodégénératives [Bordeaux] [IMN]
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorSAMIER FOUBERT, Alexandra
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorSAULNIER, Tiphaine
ORCID: 0000-0001-8551-4200
dc.contributor.authorLE TRAON, Anne Pavy
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorPROUST LIMA, Cecile
ORCID: 0000-0002-9884-955X
IDREF: 114375747
dc.contributor.authorRASCOL, Olivier
hal.structure.identifierInstitut des Maladies Neurodégénératives [Bordeaux] [IMN]
dc.contributor.authorMEISSNER, Wassilios
IDREF: 113664761
dc.date.accessioned2024-03-12T15:11:00Z
dc.date.available2024-03-12T15:11:00Z
dc.date.issued2024-02-28
dc.identifier.issn1534-6293 (Electronic) 1528-4042 (Linking)en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/188724
dc.description.abstractEnPURPOSE OF REVIEW: This review summarizes previous and ongoing neuroprotection trials in multiple system atrophy (MSA), a rare and fatal neurodegenerative disease characterized by parkinsonism, cerebellar, and autonomic dysfunction. It also describes the preclinical therapeutic pipeline and provides some considerations relevant to successfully conducting clinical trials in MSA, i.e., diagnosis, endpoints, and trial design. RECENT FINDINGS: Over 30 compounds have been tested in clinical trials in MSA. While this illustrates a strong treatment pipeline, only two have reached their primary endpoint. Ongoing clinical trials primarily focus on targeting α-synuclein, the neuropathological hallmark of MSA being α-synuclein-bearing glial cytoplasmic inclusions. The mostly negative trial outcomes highlight the importance of better understanding underlying disease mechanisms and improving preclinical models. Together with efforts to refine clinical measurement tools, innovative statistical methods, and developments in biomarker research, this will enhance the design of future neuroprotection trials in MSA and the likelihood of positive outcomes.
dc.language.isoENen_US
dc.subject.enAlpha-synuclein
dc.subject.enClinical trials
dc.subject.enDisease-modifying therapies
dc.subject.enMultiple system atrophy
dc.title.enRecent Advances in Clinical Trials in Multiple System Atrophy
dc.title.alternativeCurr Neurol Neurosci Repen_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1007/s11910-024-01335-0en_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
dc.identifier.pubmed38416311en_US
bordeaux.journalCurrent Neurology and Neuroscience Reportsen_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - UMR 1219en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.institutionINSERMen_US
bordeaux.institutionCNRS
bordeaux.teamACTIVE_BPHen_US
bordeaux.teamBIOSTAT_BPHen_US
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
hal.identifierhal-04501478
hal.version1
hal.date.transferred2024-03-12T15:11:02Z
hal.popularnonen_US
hal.audienceInternationaleen_US
hal.exporttrue
dc.rights.ccPas de Licence CCen_US
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