Adrenoleukodystrophy (ALD): treatment
Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .
Adrenoleukodystrophy (ALD): treatment. The evidence for this is not established. Fully referenced neurology checklist.
Lorenzo’s oil
- The evidence for this is not established
- It may help if started early: before the age of 6 years
- It has no effect on brain lesions or disease course
Immunotherapy
Locked: subscribe to see this section.
Statins
Locked: subscribe to see this section.
Allogenic haematopoietic stem cell transplantation
Locked: subscribe to see this section.
Haematopoietic stem cell gene therapy
Locked: subscribe to see this section.
Investigational treatments: Leriglitazone
Locked: subscribe to see this section.
References
- Engelen M, van Ballegoij WJC, Mallack EJ, et al. International recommendations for the diagnosis and management of patients with adrenoleukodystrophy: a consensus-based approach. Neurology 2022; 99:940-951.
- Mukherjee S, Newby E, Harvey JN. Adrenomyeloneuropathy in patients with `Addison's disease': genetic case analysis. J R Soc Med 2006; 99:245-249.
- van Geel BM, Assies J, Wanders RJ, Barth PG. X linked adrenoleukodystrophy: clinical presentation, diagnosis, and therapy. JNNP 1997; 63:4-14.
- Restuccia D, Di Lazzaro V, Valeriani M, et al. Neurophysiologic follow-up of long-term dietary treatment in adult-onset adrenoleukodystrophy. Neurology 1999; 52:810-816.
- Kühl JS, Suarez F, Gillett GT, et al. Long-term outcomes of allogeneic haematopoietic stem cell transplantation for adult cerebral X-linked adrenoleukodystrophy. Brain 2017; 140:953-966.
- And 5 more. Subscribe to see the full list
