Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study

Conclusions: The surviving patients function at a much higher level than untreated children or symptomatic children who underwent HSCT. These results show that early HSCT changes the natural history of this disease by improving both lifespan and functional abilities. Classification of evidence: This study provides Class IV evidence that for children with early-infantile Krabbe disease, early HSCT improves lifespan and functional abilities.
Source: Neurology - Category: Neurology Authors: Tags: Leukodystrophies, Developmental disorders, All Demyelinating disease (CNS) ARTICLE Source Type: research