Dravet syndrome is a genetic developmental and epileptic encephalopathy (DEE) often caused by a mutation in SCN1A
This is the third Rare Pediatric Disease Designation for relutrigine, adding to those granted for SCN2A and SCN8A DEEs
Praxis plans to initiate an all-DEE trial (EMERALD), inclusive of Dravet syndrome, in 1H2025
BOSTON, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for relutrigine in Dravet syndrome.