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Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses.

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Title: Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses.
Authors: Martakis K; Department of Pediatrics, University of Cologne, 50923, Cologne, Germany. Kyriakos.martakis@uk-koeln.de.; Department of Neuropediatrics, Justus Liebig University, Giessen, Germany. Kyriakos.martakis@uk-koeln.de.; Third Department of Paediatrics, Medical School, Attikon' University General Hospital, Chaidari, National and Kapodistrian University of Athens, Athens, Attica, Greece. Kyriakos.martakis@uk-koeln.de., Abreu NJ; Division of Neurogenetics, Department of Neurology, NYU Grossman School of Medicine, New York, NY, USA., Baker JJ; Edwards Family Division of Genetics and Rare Diseases, Ann and Robert H Lurie Children's Hospital of Chicago, Illinois, USA., Baker Ii PR; Department of Pediatrics, University of Colorado, Aurora, CO, USA., Billington I; IntraBio Inc, Austin, TX, USA., Burrow TA; College of Medicine, Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, AR, USA., Factor M; IntraBio Inc, Austin, TX, USA.; Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, UK., Fields T; IntraBio Inc, Austin, TX, USA.; Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, UK., Fields C; IntraBio Inc, Austin, TX, USA., Gannon JL; Division of Clinical Genetics, Children's Mercy Hospital, Kansas City, MO, USA.; Department of Pediatrics, University of Missouri-Kansas City School of Medicine, Kansas City, MO, USA., Grosso M; IntraBio Inc, Austin, TX, USA., Kerthi J; IntraBio Inc, Austin, TX, USA., Patterson MC; IntraBio Inc, Austin, TX, USA., Shayota BJ; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, UT, USA., Strupp M; Department of Neurology, Ludwig Maximilians University, Munich, Germany., Strupp L; IntraBio Inc, Austin, TX, USA., Bremova-Ert T; Department of Neurology, University Hospital Bern (Inselspital), Bern, Switzerland.
Source: Journal of neurology [J Neurol] 2026 Jul 17; Vol. 273 (8). Date of Electronic Publication: 2026 Jul 17.
Publication Type: Journal Article; Multicenter Study
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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  Data: Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses.
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  Data: <searchLink fieldCode="AU" term="%22Martakis+K%22">Martakis K</searchLink>; Department of Pediatrics, University of Cologne, 50923, Cologne, Germany. Kyriakos.martakis@uk-koeln.de.; Department of Neuropediatrics, Justus Liebig University, Giessen, Germany. Kyriakos.martakis@uk-koeln.de.; Third Department of Paediatrics, Medical School, Attikon' University General Hospital, Chaidari, National and Kapodistrian University of Athens, Athens, Attica, Greece. Kyriakos.martakis@uk-koeln.de.<br /><searchLink fieldCode="AU" term="%22Abreu+NJ%22">Abreu NJ</searchLink>; Division of Neurogenetics, Department of Neurology, NYU Grossman School of Medicine, New York, NY, USA.<br /><searchLink fieldCode="AU" term="%22Baker+JJ%22">Baker JJ</searchLink>; Edwards Family Division of Genetics and Rare Diseases, Ann and Robert H Lurie Children's Hospital of Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Baker+Ii+PR%22">Baker Ii PR</searchLink>; Department of Pediatrics, University of Colorado, Aurora, CO, USA.<br /><searchLink fieldCode="AU" term="%22Billington+I%22">Billington I</searchLink>; IntraBio Inc, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Burrow+TA%22">Burrow TA</searchLink>; College of Medicine, Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, AR, USA.<br /><searchLink fieldCode="AU" term="%22Factor+M%22">Factor M</searchLink>; IntraBio Inc, Austin, TX, USA.; Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, UK.<br /><searchLink fieldCode="AU" term="%22Fields+T%22">Fields T</searchLink>; IntraBio Inc, Austin, TX, USA.; Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, UK.<br /><searchLink fieldCode="AU" term="%22Fields+C%22">Fields C</searchLink>; IntraBio Inc, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Gannon+JL%22">Gannon JL</searchLink>; Division of Clinical Genetics, Children's Mercy Hospital, Kansas City, MO, USA.; Department of Pediatrics, University of Missouri-Kansas City School of Medicine, Kansas City, MO, USA.<br /><searchLink fieldCode="AU" term="%22Grosso+M%22">Grosso M</searchLink>; IntraBio Inc, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Kerthi+J%22">Kerthi J</searchLink>; IntraBio Inc, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Patterson+MC%22">Patterson MC</searchLink>; IntraBio Inc, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Shayota+BJ%22">Shayota BJ</searchLink>; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, UT, USA.<br /><searchLink fieldCode="AU" term="%22Strupp+M%22">Strupp M</searchLink>; Department of Neurology, Ludwig Maximilians University, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Strupp+L%22">Strupp L</searchLink>; IntraBio Inc, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Bremova-Ert+T%22">Bremova-Ert T</searchLink>; Department of Neurology, University Hospital Bern (Inselspital), Bern, Switzerland.
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