Krabbe Disease — Research Summary
Printed from RareWays (rareways.com.au) on 10 June 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Novel GALC Deletion and Paradoxical Optic Nerve Hypertrophy in Severe Infantile Krabbe Disease.
Sharma Yashu et al. — Neuropediatrics (1 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41638225/
- 2.
Spatially resolved lipids in a mouse brain model of globoid cell leukodystrophy via IR-MALDESI MSI and parallel reaction monitoring MSI.
Hunter Sierra N et al. — Analytical and bioanalytical chemistry (1 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41578040/
- 3.
Sphingolipid-neutralizing molecular therapy reduces psychosine cytotoxicity in Krabbe disease.
Begum Salma et al. — iScience (20 March 2026)
https://pubmed.ncbi.nlm.nih.gov/41743237/
- 4.
Gene therapy for Krabbe disease: evidence from mouse and canine models.
Li Yang et al. — Gene (20 February 2026)
https://pubmed.ncbi.nlm.nih.gov/41381017/
- 5.
Very late-onset Krabbe disease with concomitant dementia: case description and a critical review of the literature.
Rossi Salvatore et al. — Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology (13 February 2026)
https://pubmed.ncbi.nlm.nih.gov/41686260/
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Krabbe Disease
Krabbe disease is a rare lysosomal storage disorder caused by galactocerebrosidase (GALC) enzyme deficiency, causing rapid and fatal demyelination of the nervous system in infants. Hematopoietic stem cell transplantation can slow progression when performed before symptom onset, making newborn screening critical.
Most Recent Research
Common Questions
What is Krabbe Disease?
Krabbe disease is a rare lysosomal storage disorder caused by galactocerebrosidase (GALC) enzyme deficiency, causing rapid and fatal demyelination of the nervous system in infants. Hematopoietic stem cell transplantation can slow progression when performed before symptom onset, making newborn screening critical.
How many clinical trials are available for Krabbe Disease?
RareWays currently indexes 15 clinical trials for Krabbe Disease, of which 4 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Krabbe Disease come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is updated regularly by Rocky, RareWays' automated research engine. All articles and trials link directly to their original sources.
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This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.