Description:Â It is expected that this scientific workshop will provide
the opportunities:
1) To review the clinical course of all available presymptomatic
children transplanted for Krabbe disease, to include serial
neuroimaging, cerebrospinal fluid findings, and neurophysiologic
studies. (Is the defect central, peripheral or both? Are there
differences in outcome based on whether the child received a cord blood
transplant or allogeneic transplant? Do children fare better if
transplanted pre-term?)
2) To review the outcomes of transplanted Krabbe Disease animal models,
i.e. mouse, dog and monkey. Is the same deterioration occurring? If so,
what is the neuropathology?
3) Explore possible etiologies for the neurologic deterioration and
propose studies which may confirm or refute these theories.
Additional Information:Â Conference Web Site.