- X-ALDSafety and efficacy of leriglitazone in childhood cerebral adrenoleukodystrophy (NEXUS): an interim analysis of an open-label, phase 2/3 trial
eClinicalMedicine
2025
Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophyBrain
2024
Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2–3 trialLancet Neurology
2023
Clinical pharmacokinetics of leriglitazone and a translational approach using PBPK modeling to guide the selection of the starting dose in childrenCPT: Pharmacometrics & Systems Pharmacology
2024
The brain penetrant PPAR GAMMA agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophyScience Translational Medicine
2021