Leukodystrophy, Globoid Cell
Recent clinical, regulatory, research and industry developments relating to this disease.
Ultrastructural Characterization of the Lower Motor System in a Mouse Model of Krabbe Disease.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 1 clinical trial expected to report results, the earliest in Q4 2026.
Clinical MilestonesViewHide
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.
Leading journals1
- Scientific reports1
Leading researchers8
- Cappello V1
- Cecchini M1
- Gemmi M1
- Landi S1
- Marchetti L1
- Parlanti P1
- Piazza V1
- Tonazzini I1
Affiliations (unnormalised)1
- Center for Nanotechnology1
Reference
Authoritative identity, definition & identifiers.
An autosomal recessive metabolic disorder caused by a deficiency of GALACTOSYLCERAMIDASE leading to intralysosomal accumulation of galactolipids such as GALACTOSYLCERAMIDES and PSYCHOSINE. It is characterized by demyelination associated with large multinucleated globoid cells, predominantly involving the white matter of the central nervous system. The loss of MYELIN disrupts normal conduction of nerve impulses.
- 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.