Gonda 8-104/Joseph 1-242

Peds
Marc Patterson, M.D.
"Advances in the Diagnosis and Management of Niemann-Pick Disease, Type C"

Learning objectives:
Recognize advances in basic science that have led to improved diagnosis for Niemann-Pick disease, type C
Review the rationale for current approved and experimental therapies for Niemann-Pick disease, type C

The presenter (and/or spouse/partner) has the following relevant financial relationships to disclose:
Consultant - Actelion, Amicus, Orphazyme, Shire, Vtesse
The presenter intends to discuss off-label/investigative uses of the following commercial products/devices:
Manufacturer/Provider - Actelion; Product/Device - Miglustat