[Read more...]
Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders.
[Read more...]
The critical role of psychosine in screening, diagnosis, and monitoring of Krabbe disease.
[Read more...]
Screening for Krabbe disease: The first 2 years’ experience
[Read more...]
A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life
[Read more...]
Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months
[Read more...]
Developmental Outcomes of Cord Blood Transplantation for Krabbe Disease
[Read more...]
Intrathecal Administration of AAV/GALC Vectors in 10-11 Day old Twitcher Mice Improves Survival and is Enhanced by Bone Marrow Transplant.
[Read more...]
Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice.
[Read more...]
Psychosine, a marker of Krabbe phenotype and treatment effect.
[Read more...]