Publications by authors named "Caroline Sevin"

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Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
Florian Eichler, Christine N Duncan, Patricia L Musolino, Troy C Lund, Ashish O Gupta, Satiro De Oliveira, Adrian J Thrasher, Patrick Aubourg, Jörn-Sven Kühl, Daniel J Loes

N Engl J Med· October 2024

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Encapsulated cells as an enzyme replacement therapy for metachromatic leukodystrophy.
Emilie Audouard, Florine Chereau, Camille Lupiet, Charlotte Mansat, Lucie Roger, Caroline Sevin

J Control Release· December 2025

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Consensus-Based Expert Recommendations for Diagnosis and Clinical Management of Vanishing White Matter.
Romy J van Voorst, Daphne H Schoenmakers, Joshua L Bonkowsky, Adeline Vanderver, Ingeborg Krägeloh-Mann, Caroline Sevin

Neurology· December 2025

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ARSA Variants Associated With Cognitive Decline and Long-Term Preservation of Motor Function in Metachromatic Leukodystrophy.
Shanice Beerepoot, Daphne H Schoenmakers, Francesca Fumagalli, Samuel Groeschel, Ludger Schöls, Caroline Sevin

J Inherit Metab Dis· September 2025

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Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy.
Daphne H Schoenmakers, Marije A B C Asbreuk, Tamara Martin, Mareen Datema, Shanice Beerepoot, Caroline Sevin

Eur J Paediatr Neurol· July 2025

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Metachromatic Leukodystrophy: New Therapy Advancements and Emerging Research Directions.
Marije A B C Asbreuk, Daphne H Schoenmakers, Laura Ann Adang, Shanice Beerepoot, Caroline Bergner, Caroline Sevin

Neurology· July 2025

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Safety and efficacy of leriglitazone in childhood cerebral adrenoleukodystrophy (NEXUS): an interim analysis of an open-label, phase 2/3 trial.
Ángeles García-Cazorla, Caroline Sevin, Juliana Ribeiro Constante, Elise Yazbeck, Hendrik Rosewich

EClinicalMedicine· June 2025

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Improving paediatric movement disorders care: Insights on rating scales utilization and clinical practice.
María Eugenia Amato, Alejandra Darling, Lucie Stovickova, Stephen Attard, Hendriekje Eggink, Caroline Sevin

Eur J Paediatr Neurol· September 2024

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Hematopoietic stem cell transplantation in leukodystrophies.
Caroline Sevin, Fanny Mochel

Handb Clin Neurol· September 2024

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Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States.
Laura A Adang, Joshua L Bonkowsky, Jaap Jan Boelens, Eric Mallack, Rebecca Ahrens-Nicklas, Caroline Sevin

Cytotherapy· July 2024

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Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy.
Emilie Audouard, Nicolas Khefif, Charlotte Mansat, Océane Nelcha, Elena-Gaia Banchi, Caroline Sevin

Mol Ther Methods Clin Dev· June 2024

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Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.
Lucia Laugwitz, Daphne H Schoenmakers, Laura A Adang, Stefanie Beck-Woedl, Caroline Bergner, Caroline Sevin

Eur J Paediatr Neurol· March 2024

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Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.
Daphne H Schoenmakers, Fanny Mochel, Laura A Adang, Jaap-Jan Boelens, Valeria Calbi, Caroline Sevin

Orphanet J Rare Dis· February 2024

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Gene therapy for neurodegenerative disorders in children: dreams and realities.
Odile Boespflug-Tanguy, Caroline Sevin, Francoise Piguet

Arch Pediatr· November 2023

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Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden of disease - A population-based study.
Caroline Sevin, Samira Hatteb, Aurore Clément, Fabrizia Bignami, Louis Chillotti

Orphanet J Rare Dis· August 2023

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Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study.
Cécile Freihuber, Bahia Dahmani-Rabehi, Anaïs Brassier, Pierre Broué, Claude Cances, Caroline Sevin

Orphanet J Rare Dis· July 2023

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Prospective Multicenter Validation of a Simple Blood Test for the Diagnosis of Glut1 Deficiency Syndrome.
Fanny Mochel, Domitille Gras, Marie-Pierre Luton, Manon Nizou, Donatella Giovannini, Caroline Sevin

Neurology· June 2023

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A Retrospective Multicentric Study of 34 Patients with Niemann-Pick Type C Disease and Early Liver Involvement in France.
Antoine Gardin, Charlotte Mussini, Bénédicte Héron, Manuel Schiff, Anaïs Brassier, Caroline Sevin

J Pediatr· March 2023

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[Inborn error of metabolism and allogenic hematopoietic cell transplantation: Guidelines from the SFGM-TC].
Charlotte Jubert, Eva De Berranger, Martin Castelle, Jean-Hugues Dalle, Marie Ouachee-Chardin, Caroline Sevin

Bull Cancer· February 2023

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International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach.
Marc Engelen, Wouter J C van Ballegoij, Eric James Mallack, Keith P Van Haren, Wolfgang Köhler, Caroline Sevin

Neurology· November 2022

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Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy.
F Eichler, Caroline Sevin, M Barth, F Pang, K Howie

Orphanet J Rare Dis· October 2022

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An international study of caregiver-reported burden and quality of life in metachromatic leukodystrophy.
Caroline Sevin, Magalie Barth, Alexandra Wilds, Abena Afriyie, Markus Walz

Orphanet J Rare Dis· September 2022

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Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy.
Robert Chiesa, Jaap Jan Boelens, Christine N Duncan, Jörn-Sven Kühl, Caroline Sevin

Blood Adv· March 2022

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Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi).
Daphne H Schoenmakers, Shanice Beerepoot, Sibren van den Berg, Laura Adang, Annette Bley, Caroline Sevin

Orphanet J Rare Dis· February 2022

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Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement.
Caroline Sevin, Kumaran Deiva

Front Mol Biosci· September 2021

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