Safety and efficacy of leriglitazone in childhood cerebral adrenoleukodystrophy (NEXUS): an interim analysis of an open-label, phase 2/3 trialResearch in context

Safety and efficacy of leriglitazone in childhood cerebral adrenoleukodystrophy (NEXUS): an interim analysis of an open-label, phase 2/3 trialResearch in context

Summary: Background: Cerebral adrenoleukodystrophy rapidly progresses in approximately 90% of untreated patients. Current treatment, haematopoietic stem-cell transplantation (HSCT), is associated with high morbidity and is not widely available. Lower risk treatments that can be administered immedia...

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Main Authors: Ángeles García-Cazorla, Caroline Sevin, Juliana Ribeiro Constante, Elise Yazbeck, Hendrik Rosewich, Sandra Jimenez, Gloria Chia-Yi Chiang, Otto Rapalino, Paul Caruso, Daniel Balentine, Karl G. Helmer, Seth Bennett, Marco Emanuele, Laura Rodriguez-Pascau, Pilar Pizcueta, Guillem Pina, Anna Vilà, Maria Rovira, Adriana Mantilla, Uwe Meya, Arun Mistry, María Pascual, Sílvia Pascual, Marc Martinell, Patricia L. Musolino, Eric Mallack
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:EClinicalMedicine
Subjects:
Cerebral adrenoleukodystrophy
Paediatric adrenoleukodystrophy
PPARγ agonist
Online Access:http://www.sciencedirect.com/science/article/pii/S258953702500197X
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