Krabbe Disease Latest Advances

Durable Global Correction of CNS and PNS and Lifespan Rescue in Murine Globoid Cell Leukodystrophy via AAV9-Mediated Monotherapy.

Journal: Cells

Published: November 18, 2025

Abnormal Splicing of GALC Transcripts Underlies Unusual Cases of Krabbe Disease.

Journal: Biomedicines

Published: November 12, 2025

Correction: Generalized tonic-clonic seizures as the initial symptom of late-onset Krabbe disease: a Case Report.

Journal: Frontiers in behavioral neuroscience

Published: November 06, 2025

Caregiver-reported disease burden in Krabbe disease: evaluating outcomes of hematopoietic stem cell transplantation.

Journal: Orphanet journal of rare diseases

Published: November 03, 2025

Advanced biomaterials for rare Krabbe disease: galactocerebrosidase scaffolds in demyelinating lesions.

Journal: Annals of medicine and surgery (2012)

Published: October 21, 2025

A case of adult-onset Krabbe disease diagnosed by galactocerebrosidase gene mutations, presenting with an atypical phenotype

Journal: Rinsho shinkeigaku = Clinical neurology

Published: October 19, 2025

Gene therapy for Krabbe disease: evidence from mouse and canine models.

Journal: Gene

Published: October 12, 2025

Genetic and Clinical Characteristics of Chinese Adult Patients With Krabbe Disease.

Journal: CNS neuroscience & therapeutics

Published: September 25, 2025

CRISPR-mediated correction of GALC mutations in Krabbe disease neural models - opportunities and risks.

Journal: Annals of medicine and surgery (2012)

Published: September 21, 2025

Ceramide levels predict clinical severity in adult-onset Krabbe disease independent of extensive white matter hyperintensities.

Journal: Neurobiology of disease

Published: September 15, 2025

Ceramide levels predict clinical severity in adult-onset Krabbe disease independent of extensive white matter hyperintensities.

Journal: Neurobiology of disease

Published: September 15, 2025

Impaired docking and recycling of synaptic vesicles in inherited lysosomal sphingolipidoses.

Journal: Cell communication and signaling : CCS

Published: September 10, 2025