Rare Diseases
News from NORD
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FDA/CDC
FDA approves Epidiolex for Lennox-Gastaut syndrome and Dravet syndrome
The approval, the first for a marijuana-derived pharmaceutical product, falls in line with the unanimous recommendation of an FDA advisory panel...
Conference Coverage
Myelin antibody predicts ADEM relapse
LOS ANGELES – Could results help to guide decisions on immunosuppressive treatment?
Conference Coverage
Guidance coming for mTOR inhibitors in infantile TSC
LOS ANGELES – Survey aimed to describe the wide variation in their off-label use, particularly in infants.