New therapeutic strategies for Lafora disease: Evaluation of the safety, efficacy, pharmacokinetics and metabolomic profile of intravenous VAL-1221 treatment.
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| Title: | New therapeutic strategies for Lafora disease: Evaluation of the safety, efficacy, pharmacokinetics and metabolomic profile of intravenous VAL-1221 treatment. |
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| Authors: | Muccioli L; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy. Electronic address: lorenzo.muccioli@gmail.com., Tappatà M; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Farolfi A; Nuclear Medicine, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy., Singh PK; Department of Biochemistry and Molecular Biology, University of Florida, College of Medicine, Gainesville, FL, USA., Pasini E; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Mazzone S; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Esposito E; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Motta L; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., D'Agati OM; Department of Biochemistry and Molecular Biology, University of Florida, College of Medicine, Gainesville, FL, USA., Angeloni S; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy., Caravelli A; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Cancellerini C; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy., Minardi R; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Pizzi E; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy., Carelli V; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy., Tonon C; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy., Fiori J; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Chemistry, University of Bologna, Bologna, Italy., Gentry MS; Department of Biochemistry and Molecular Biology, University of Florida, College of Medicine, Gainesville, FL, USA., Vignatelli L; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Michelucci R; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy., Bisulli F; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy. |
| Corporate Authors: | DEFEAT-LD study group |
| Source: | Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics [Neurotherapeutics] 2026 Jan; Vol. 23 (1), pp. e00808. Date of Electronic Publication: 2025 Nov 30. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Elsevier Inc. on behalf of American Society for Experimental NeuroTherapeutics Country of Publication: United States NLM ID: 101290381 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-7479 (Electronic) Linking ISSN: 18787479 NLM ISO Abbreviation: Neurotherapeutics Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 41326286 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: New therapeutic strategies for Lafora disease: Evaluation of the safety, efficacy, pharmacokinetics and metabolomic profile of intravenous VAL-1221 treatment. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Muccioli+L%22">Muccioli L</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy. Electronic address: lorenzo.muccioli@gmail.com.<br /><searchLink fieldCode="AU" term="%22Tappatà+M%22">Tappatà M</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Farolfi+A%22">Farolfi A</searchLink>; Nuclear Medicine, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Singh+PK%22">Singh PK</searchLink>; Department of Biochemistry and Molecular Biology, University of Florida, College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Pasini+E%22">Pasini E</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Mazzone+S%22">Mazzone S</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Esposito+E%22">Esposito E</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Motta+L%22">Motta L</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22D'Agati+OM%22">D'Agati OM</searchLink>; Department of Biochemistry and Molecular Biology, University of Florida, College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Angeloni+S%22">Angeloni S</searchLink>; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Caravelli+A%22">Caravelli A</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Cancellerini+C%22">Cancellerini C</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Minardi+R%22">Minardi R</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Pizzi+E%22">Pizzi E</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Carelli+V%22">Carelli V</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Tonon+C%22">Tonon C</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Fiori+J%22">Fiori J</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Chemistry, University of Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Gentry+MS%22">Gentry MS</searchLink>; Department of Biochemistry and Molecular Biology, University of Florida, College of Medicine, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Vignatelli+L%22">Vignatelli L</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Michelucci+R%22">Michelucci R</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Bisulli+F%22">Bisulli F</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna (Full Member of the ERN EpiCARE), Bologna, Italy; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy. – Name: AuthorCorporate Label: Corporate Authors Group: Au Data: <searchLink fieldCode="CA" term="%22DEFEAT-LD+study+group%22">DEFEAT-LD study group</searchLink> – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101290381%22">Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics</searchLink> [Neurotherapeutics] 2026 Jan; Vol. 23 (1), pp. e00808. <i>Date of Electronic Publication: </i>2025 Nov 30. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier+Inc%2E+on+behalf+of+American+Society+for+Experimental+NeuroTherapeutics%22">Elsevier Inc. on behalf of American Society for Experimental NeuroTherapeutics </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101290381 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1878-7479 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2218787479%22">18787479 </searchLink><i>NLM ISO Abbreviation: </i>Neurotherapeutics <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41326286 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1016/j.neurot.2025.e00808 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: e00808 Titles: – TitleFull: New therapeutic strategies for Lafora disease: Evaluation of the safety, efficacy, pharmacokinetics and metabolomic profile of intravenous VAL-1221 treatment. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Muccioli L – PersonEntity: Name: NameFull: Tappatà M – PersonEntity: Name: NameFull: Farolfi A – PersonEntity: Name: NameFull: Singh PK – PersonEntity: Name: NameFull: Pasini E – PersonEntity: Name: NameFull: Mazzone S – PersonEntity: Name: NameFull: Esposito E – PersonEntity: Name: NameFull: Motta L – PersonEntity: Name: NameFull: D'Agati OM – PersonEntity: Name: NameFull: Angeloni S – PersonEntity: Name: NameFull: Caravelli A – PersonEntity: Name: NameFull: Cancellerini C – PersonEntity: Name: NameFull: Minardi R – PersonEntity: Name: NameFull: Pizzi E – PersonEntity: Name: NameFull: Carelli V – PersonEntity: Name: NameFull: Tonon C – PersonEntity: Name: NameFull: Fiori J – PersonEntity: Name: NameFull: Gentry MS – PersonEntity: Name: NameFull: Vignatelli L – PersonEntity: Name: NameFull: Michelucci R – PersonEntity: Name: NameFull: Bisulli F IsPartOfRelationships: – BibEntity: Dates: – D: 01 M: 01 Text: 2026 Jan Type: published Y: 2026 Identifiers: – Type: issn-electronic Value: 1878-7479 Numbering: – Type: volume Value: 23 – Type: issue Value: 1 Titles: – TitleFull: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics Type: main |
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