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 |
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