Disease

Adrenoleukodystrophy1, 2

Gene

ABCD1

Inheritance XL
Current Treatments Hematopoietic stem cell transplantation (HSCT) - bone marrow transplant, Elivaldogene Autotemcel
Evidence for Treatment guideline3
Non-Molecular Testing Availability yes
Non-Molecular Test plasma very long chain fatty acids4
ClinGen https://actionability.clinicalgenome.org/ac/Adult/ui/stg2SummaryRpt?doc=AC117
Disease Frequency (per 100,000) 3.51
Genes Included in Disease Frequency ABCD1
Experimental / Investigational Therapies https://clinicaltrials.gov/ct2/results?cond=Adrenoleukodystrophy&term=&cntry=&state=&city=&dist=
Last Updated: March 5, 2026
RX Genes
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