N-acetyl-L-cysteine improves outcome of advanced cerebral adrenoleukodystrophy

Abstract Hematopoietic stem cell transplantation as a treatment for childhood cerebral adrenoleukodystrophy (ALD) has historically only been successful in early disease. As ALD is associated with oxidative damage, we reasoned that adjunctive therapy with an antioxidant agent, N-acetyl-L-cysteine (NAC), may provide protection from rapid neurologic decline in boys with advanced cerebral disease. We report three…

Combo of Antioxidants a Potential Therapy for Adrenomyeloneuropathy, Trial Suggests

Targeting oxidative stress with antioxidants holds promise to improve symptoms of adrenomyeloneuropathy (AMN), a pilot Phase 2 study suggests. The results show that treatment with a combo of antioxidants decreases inflammation and enhances patients’ walking capacity. The study, “Biomarker identification, Safety and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy: a Phase II Pilot study,” was published…