NOVEL MICRO-RNA THERAPY FOR TREATMENT OF DRAVET SYNDROME, A PEDIATRIC ORPHAN DISEASE
April 24, 2014
Dravet syndrome (DS), also known as severe myoclonic epilepsy of infancy (SMEI), is a genetic and rare form of treatment-refractory epilepsy that begins in infancy. DS incidence is estimated at 1/30,000 with about 20,000-40,000 patients worldwide. The long-term prognosis for DS patients …