Parent-reported disease burden in Krabbe disease: Evaluating outcomes of hematopoietic stem cell transplant

Krabbe disease (KD) is a rapidly progressive neurodegenerative disorder caused by β-galactocerebrosidase deficiency. The only approved disease modifying therapy for infantile KD (IKD) and late-infantile KD (LIKD) is hematopoietic stem cell transplantation (HSCT) when performed in pre-symptomatic stages.