Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease

Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders

(2020) Mark T. Vander Lugt et al.   Blood Advances

Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months

(2019) Maria L. Beltran-Quintero et al.   Orphanet Journal of Rare Diseases

Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease

(2018) Heather Allewelt et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Hematopoietic Stem Cell Transplantation in Late-Onset Krabbe Disease: No Evidence of Worsening Demyelination and Axonal Loss 4 Years Post-allograft

(2018) Cornelia Laule et al.   JOURNAL OF NEUROIMAGING

A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life

(2018) Nicholas Bascou et al.   Orphanet Journal of Rare Diseases

Psychosine, a marker of Krabbe phenotype and treatment effect

(2017) M.L. Escolar et al.   MOLECULAR GENETICS AND METABOLISM

Developmental outcomes of cord blood transplantation for Krabbe disease

(2017) Matthew D. Wright et al.   NEUROLOGY

Clinical management of Krabbe disease

(2016) Maria L. Escolar et al.   JOURNAL OF NEUROSCIENCE RESEARCH

A Novel Reduced-Intensity Conditioning Regimen for Unrelated Umbilical Cord Blood Transplantation in Children with Nonmalignant Diseases

(2013) Suhag H. Parikh et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Determination of psychosine concentration in dried blood spots from newborns that were identified via newborn screening to be at risk for Krabbe disease

(2013) Wei-Lien Chuang et al.   CLINICA CHIMICA ACTA

Early Infantile Krabbe Disease: Results of the World-Wide Krabbe Registry

(2012) Patricia K. Duffner et al.   PEDIATRIC NEUROLOGY

Later Onset Phenotypes of Krabbe Disease: Results of the World-Wide Registry

(2012) Patricia K. Duffner et al.   PEDIATRIC NEUROLOGY

Identification and characterization of 15 novel GALC gene mutations causing Krabbe disease

(2010) Barbara Tappino et al.   HUMAN MUTATION

Diffusion Tensor Imaging Detects Abnormalities in the Corticospinal Tracts of Neonates with Infantile Krabbe Disease

(2009) M.L. Escolar et al.   AMERICAN JOURNAL OF NEURORADIOLOGY

Methods for assessing neurodevelopment in lysosomal storage diseases and related disorders: a multidisciplinary perspective

(2008) Holly R Martin et al.   ACTA PAEDIATRICA

Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes

(2008) V. K. Prasad et al.   BLOOD