Current and Future Pharmacological Treatment Strategies in X-Linked Adrenoleukodystrophy

Abstract DMutations in the ABCD1 gene cause the clinical spectrum of the neurometabolic disorder X-linked adrenoleukodystrophy/adrenomyeloneuropathy (X-ALD/AMN). Currently, the most efficient therapeutic opportunity for patients with the cerebral form of X-ALD is hematopoietic stem cell transplantation and possibly gene therapy of autologous hematopoietic stem cells. Both treatments, however, are only accessible to a subset of…

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…