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X-linked cerebral adrenoleukodystrophy (ALD) - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

X-linked cerebral adrenoleukodystrophy

MONDO:0010247

Also known as:ALDALD childhood cerebral formAddison disease and cerebral sclerosis

Find Clinical TrialsTrials

Overview

A peroxisomal disease characterized by severe inflammatory demyelination in the brain, and often associated with adrenal insufficiency.

Treatments1 FDA approved, incl. 1 gene therapy

Clinical Trials19 registered · 10 recruiting

Diagnosis

Biomarker and diagnostic research for X-linked cerebral adrenoleukodystrophy has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

1 FDA-approved treatment is available for X-linked cerebral adrenoleukodystrophy, including elivaldogene autotemcel (Skysona, approved 2022).

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

Prognosis

Estimated prevalence: Unknown (Unknown prevalence).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

19 clinical trials registered, 10 recruiting. Interventions under study include other interventions, drug therapy, gene therapy, and biologic therapy. Pipeline includes 1 PHASE4, 1 PHASE3, 2 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06224413](https://clinicaltrials.gov/study/NCT06224413)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 10:22 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:139396

GARD

Genetic and Rare Diseases Info Center

GARD:9412

Questions for Your Care Team

→What are the eligibility criteria and monitoring requirements for Skysona?
→Are any of the 19 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Research Type	Count	% of Total
Patient case studies	3	30%
Laboratory research	3	30%
Other research	2	20%
Testing and diagnosis research	1	10%
Research summaries	1	10%

Kisho

X-linked cerebral adrenoleukodystrophy

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Nezglyal receives positive CHMP opinion supporting treatment access for boys with cALD

The complex lipidome as a driver of tissue-specific pathology in adrenoleukodystrophy.

Profiling of fatty acids and lipids in animal and human tissues yields new leads for disease progression biomarkers of X-linked adrenoleukodystrophy.

Pediatric X-linked adrenoleukodystrophy: phenotypes, variants, and HSCT outcomes.

Gene Therapy Pricing | Rare Disease Access Barriers | Trial Friend

X-linked cerebral adrenoleukodystrophy

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Nezglyal receives positive CHMP opinion supporting treatment access for boys with cALD

The complex lipidome as a driver of tissue-specific pathology in adrenoleukodystrophy.

Profiling of fatty acids and lipids in animal and human tissues yields new leads for disease progression biomarkers of X-linked adrenoleukodystrophy.

Pediatric X-linked adrenoleukodystrophy: phenotypes, variants, and HSCT outcomes.

Gene Therapy Pricing | Rare Disease Access Barriers | Trial Friend