Adrenoleukodystrophy
Recent clinical, regulatory, research and industry developments relating to this disease.
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 2 clinical trials expected to report results, the earliest in Q4 2026.
- Q4 2026Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells
- Q2 2027A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Clinical MilestonesViewHide
- 2025-11-17A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral AdrenoleukodystrophyResults expected Q2 2027
- 2025-09-08Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like CellsResults expected Q4 2026
- 2026-01-05ClinicalMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGPrimary completion
- 2025-11-17ClinicalA Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral AdrenoleukodystrophyResults expected Q2 2027
- 2025-09-08ClinicalAugmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like CellsResults expected Q4 2026
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes3
- Genetic Therapy1
- Genetic Vectors1
- Hematopoietic Stem Cell Transplantation1
Leading journals2
- International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience1
- Science (New York, N.Y.)1
Leading researchers8
- Abel U1
- Aubourg P1
- Audit M1
- Bartholomae CC1
- Bellesme C1
- Blanche S1
- Bougnères P1
- Caccavelli L1
Affiliations (unnormalised)3
- Hugo W Moser Research Institute1
- Royal Women's Hospital1
- University Paris-Descartes1
Disease biology
Key proteins & gene products studied in this disease. Number shows shared papers.
Reference
Authoritative identity, definition & identifiers.
An X-linked recessive disorder characterized by the accumulation of saturated very long chain fatty acids in the LYSOSOMES of ADRENAL CORTEX and the white matter of CENTRAL NERVOUS SYSTEM. This disease occurs almost exclusively in the males. Clinical features include the childhood onset of ATAXIA; NEUROBEHAVIORAL MANIFESTATIONS; HYPERPIGMENTATION; ADRENAL INSUFFICIENCY; SEIZURES; MUSCLE SPASTICITY; and DEMENTIA. The slowly progressive adult form is called adrenomyeloneuropathy. The defective gene ABCD1 is located at Xq28, and encodes the adrenoleukodystrophy protein (ATP-BINDING CASSETTE TRANSPORTERS).
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.