PRAX - Praxis Precision's pediatric epilepsy therapies nab Rare Pediatric and Orphan Drug tags in U.S.

The FDA has designated Rare Pediatric and Orphan Drug status to Praxis Precision Medicines's ([[PRAX]] +3.4%) PRAX-222, for the treatment of SCN2A developmental and epileptic encephalopathy (SCN2A-DEE). as well as Rare Pediatric tag for PRAX-562 for SCN2A-DEE and SCN8A developmental and epileptic encephalopathy (SCN8A-DEE).PRAX-562 is a selective small molecule and is the first persistent sodium current blocker in development for the treatment of a wide range of rare CNS disorders.PRAX-222 is an antisense oligonucleotide that is designed to lower the expression levels of the protein encoded by the SCN2A gene in patients with SCN2A gain-of-function epilepsy.Press Release

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Praxis Precision's pediatric epilepsy therapies nab Rare Pediatric and Orphan Drug tags in U.S.