Open-label evaluation of oral trehalose in patients with neuronal ceroid lipofuscinoses.
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| Title: | Open-label evaluation of oral trehalose in patients with neuronal ceroid lipofuscinoses. |
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| Authors: | Della Vecchia S; Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA), University of Florence, Viale Pieraccini, 6, 50139, Florence, Italy.; IRCCS Stella Maris Foundation, Via Dei Giacinti 2, 56128, Pisa, Italy., Gammaldi N; Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA), University of Florence, Viale Pieraccini, 6, 50139, Florence, Italy.; IRCCS Stella Maris Foundation, Via Dei Giacinti 2, 56128, Pisa, Italy., Ricca I; IRCCS Stella Maris Foundation, Via Dei Giacinti 2, 56128, Pisa, Italy., Mero S; IRCCS Stella Maris Foundation, Via Dei Giacinti 2, 56128, Pisa, Italy., Doccini S; IRCCS Stella Maris Foundation, Via Dei Giacinti 2, 56128, Pisa, Italy., Ardissone A; Child Neurology Unit, Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Besta, Milan, Italy., Bagnoli S; Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA), University of Florence, Viale Pieraccini, 6, 50139, Florence, Italy.; IRCCS Fondazione Don Carlo Gnocchi, Florence, Italy., Battini R; IRCCS Stella Maris Foundation, Via Dei Giacinti 2, 56128, Pisa, Italy.; Department of Clinical and Experimental Medicine, University of Pisa, 56126, Pisa, Italy., Colombi E; Child Neuropsychiatric Unit, ASL CN2 Alba-Bra, Alba, Italy., Favaro J; Neurology and Neurophysiology Unit, Department of Women's and Children's Health, Padua University Hospital, 35128, Padua, Italy., Furlan R; Clinical Neuroimmunology Unit, Institute of Experimental Neurology (INSpe), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita E Salute San Raffaele University, Milan, Italy., Giordano L; Paediatric Neurology and Psychiatry Unit, Spedali Civili Children's Hospital, University of Brescia, Brescia, Italy., Ingannato A; Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA), University of Florence, Viale Pieraccini, 6, 50139, Florence, Italy., Mandelli A; Clinical Neuroimmunology Unit, Institute of Experimental Neurology (INSpe), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy., Manzoni FMP; Child Neurology and Rehabilitation Service, Regional Hospital of Bolzano, Bolzano, Italy., Milito G; Paediatric Neurology and Psychiatry Unit, Spedali Civili Children's Hospital, University of Brescia, Brescia, Italy., Moroni I; Child Neurology Unit, Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Besta, Milan, Italy., Nacmias B; Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA), University of Florence, Viale Pieraccini, 6, 50139, Florence, Italy.; IRCCS Fondazione Don Carlo Gnocchi, Florence, Italy., Nardocci N; Child Neurology Unit, Department of Pediatric Neuroscience, Fondazione IRCCS Istituto Neurologico Besta, Milan, Italy., Parmeggiani L; Child Neurology and Rehabilitation Service, Regional Hospital of Bolzano, Bolzano, Italy., Pezzini F; Department of Surgery, Dentistry, Paediatrics and Gynaecology (Child Neurology and Psychiatry), University of Verona, 37134, Verona, Italy., Pietrafusa N; Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Sartori S; Neurology and Neurophysiology Unit, Department of Women's and Children's Health, Padua University Hospital, 35128, Padua, Italy., Specchio N; Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Trivisano M; Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Ets ACL; Associazione Nazionale CeroidoLipofuscinosi Onlus, Via Oberdan 3, 76015, Trinitapoli, Italy., Simonati A; Department of Surgery, Dentistry, Paediatrics and Gynaecology (Child Neurology and Psychiatry), University of Verona, 37134, Verona, Italy., Santorelli FM; IRCCS Stella Maris Foundation, Via Dei Giacinti 2, 56128, Pisa, Italy. filippo3364@gmail.com. |
| Corporate Authors: | A-NCL ETS Group |
| Source: | Journal of neurology [J Neurol] 2025 Jan 07; Vol. 272 (1), pp. 94. Date of Electronic Publication: 2025 Jan 07. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Springer-Verlag Country of Publication: Germany NLM ID: 0423161 Publication Model: Electronic Cited Medium: Internet ISSN: 1432-1459 (Electronic) Linking ISSN: 03405354 NLM ISO Abbreviation: J Neurol Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1432-1459 |
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| DOI: | 10.1007/s00415-024-12790-7 |
