A peroxisomal disease characterized by severe inflammatory demyelination in the brain, and often associated with adrenal insufficiency.
Comprehensive, easy-to-understand information about this condition
How we create this content →The documentation surrounding X-ALD is limited due to its rarity and the complexity of its clinical features. Because X-ALD affects fewer than 1 in 20,000 individuals, systematic clinical studies have been scarce. Additionally, the lack of identified genes and established inheritance patterns further complicates the understanding of this condition, making it difficult to gather comprehensive clinical data.
To navigate your care for X-ALD, consider consulting a neurologist with expertise in leukodystrophies or metabolic disorders. Additionally, genetic counseling may be beneficial, especially given the unknowns regarding inheritance. While no specific patient organizations exist for X-ALD, you can find resources at GARD (rarediseases.info.nih.gov) and NORD (rarediseases.org). Participating in clinical trials may also provide access to cutting-edge treatments and contribute to the understanding of the disease.
There are currently 20 active clinical trials investigating various aspects of X-ALD, which provides hope for future treatment options. One FDA-approved treatment is elivaldogene autotemcel (Skysona). Additionally, several orphan drugs are in development, including compounds targeting the underlying mechanisms of the disease. For more information on ongoing trials, you can visit ClinicalTrials.gov at https://clinicaltrials.gov/search?cond=X-linked%20cerebral%20adrenoleukodystrophy.
Actionable guidance for navigating care for X-linked cerebral adrenoleukodystrophy
To navigate your care for X-ALD, consider consulting a neurologist with expertise in leukodystrophies or metabolic disorders. Additionally, genetic counseling may be beneficial, especially given the unknowns regarding inheritance. While no specific patient organizations exist for X-ALD, you can find resources at GARD (rarediseases.info.nih.gov) and NORD (rarediseases.org). Participating in clinical trials may also provide access to cutting-edge treatments and contribute to the understanding of the disease.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
The documentation surrounding X-ALD is limited due to its rarity and the complexity of its clinical features. Because X-ALD affects fewer than 1 in 20,000 individuals, systematic clinical studies have been scarce. Additionally, the lack of identified genes and established inheritance patterns further complicates the understanding of this condition, making it difficult to gather comprehensive clinical data.
To navigate your care for X-ALD, consider consulting a neurologist with expertise in leukodystrophies or metabolic disorders. Additionally, genetic counseling may be beneficial, especially given the unknowns regarding inheritance. While no specific patient organizations exist for X-ALD, you can find resources at GARD (rarediseases.info.nih.gov) and NORD (rarediseases.org). Participating in clinical trials may also provide access to cutting-edge treatments and contribute to the understanding of the disease.
There are currently 20 active clinical trials investigating various aspects of X-ALD, which provides hope for future treatment options. One FDA-approved treatment is elivaldogene autotemcel (Skysona). Additionally, several orphan drugs are in development, including compounds targeting the underlying mechanisms of the disease. For more information on ongoing trials, you can visit ClinicalTrials.gov at https://clinicaltrials.gov/search?cond=X-linked%20cerebral%20adrenoleukodystrophy.
Actionable guidance for navigating care for X-linked cerebral adrenoleukodystrophy
To navigate your care for X-ALD, consider consulting a neurologist with expertise in leukodystrophies or metabolic disorders. Additionally, genetic counseling may be beneficial, especially given the unknowns regarding inheritance. While no specific patient organizations exist for X-ALD, you can find resources at GARD (rarediseases.info.nih.gov) and NORD (rarediseases.org). Participating in clinical trials may also provide access to cutting-edge treatments and contribute to the understanding of the disease.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
The documentation surrounding X-ALD is limited due to its rarity and the complexity of its clinical features. Because X-ALD affects fewer than 1 in 20,000 individuals, systematic clinical studies have been scarce. Additionally, the lack of identified genes and established inheritance patterns further complicates the understanding of this condition, making it difficult to gather comprehensive clinical data.
To navigate your care for X-ALD, consider consulting a neurologist with expertise in leukodystrophies or metabolic disorders. Additionally, genetic counseling may be beneficial, especially given the unknowns regarding inheritance. While no specific patient organizations exist for X-ALD, you can find resources at GARD (rarediseases.info.nih.gov) and NORD (rarediseases.org). Participating in clinical trials may also provide access to cutting-edge treatments and contribute to the understanding of the disease.
There are currently 20 active clinical trials investigating various aspects of X-ALD, which provides hope for future treatment options. One FDA-approved treatment is elivaldogene autotemcel (Skysona). Additionally, several orphan drugs are in development, including compounds targeting the underlying mechanisms of the disease. For more information on ongoing trials, you can visit ClinicalTrials.gov at https://clinicaltrials.gov/search?cond=X-linked%20cerebral%20adrenoleukodystrophy.
Actionable guidance for navigating care for X-linked cerebral adrenoleukodystrophy
To navigate your care for X-ALD, consider consulting a neurologist with expertise in leukodystrophies or metabolic disorders. Additionally, genetic counseling may be beneficial, especially given the unknowns regarding inheritance. While no specific patient organizations exist for X-ALD, you can find resources at GARD (rarediseases.info.nih.gov) and NORD (rarediseases.org). Participating in clinical trials may also provide access to cutting-edge treatments and contribute to the understanding of the disease.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
AI-curated news mentioning X-linked cerebral adrenoleukodystrophy
Updated Feb 10, 2026
The Grey Zone Project introduces a risk-based classification system for ABCD1 variants in X-Linked Adrenoleukodystrophy, enhancing the understanding of genetic variations. This initiative aims to improve patient management and treatment strategies for affected individuals.
A new study characterizes the phenotypic features and natural history of adrenoleukodystrophy in adults, providing valuable insights into the disease's progression. This research enhances understanding of the condition, which may inform future therapeutic strategies.
The article discusses the potential of lentiviral-based gene therapy for treating rare genetic diseases affecting the brain and spinal cord, including X-linked adrenoleukodystrophy, metachromatic leukodystrophy, and mucopolysaccharidoses. It highlights the aim of improving enzyme bioavailability and correcting neuropathological phenotypes.