Praxis Precision Medicines Announces Rare Pediatric Disease Designation Granted For Relutrigine In Dravet Syndrome; Praxis Plans To Initiate An All-DEE Trial (EMERALD), Inclusive Of Dravet Syndrome, In 1H2025
Praxis Precision Medicines Announces Rare Pediatric Disease Designation Granted For Relutrigine In Dravet Syndrome; 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.Dravet syndrome is a genetic developmental and epileptic encephalopathy (DEE) often caused by a mutation in SCN1A
德拉維特綜合症是一種遺傳性發育性癲癇腦病（DEE），通常是由SCN1A的突變引起的。
This is the third Rare Pediatric Disease Designation for relutrigine, adding to those granted for SCN2A and SCN8A DEEs
這是relutrigine獲得的第三個罕見兒科疾病指定，之前已有針對SCN2A和SCN8A DEE的授權。
Praxis plans to initiate an all-DEE trial (EMERALD), inclusive of Dravet syndrome, in 1H2025
Praxis計劃在2025年上半年啓動一項涵蓋所有DEE的試驗（EMERALD），包括德拉維特綜合症。
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.
波士頓，2024年12月18日（環球新聞網） -- Praxis Precision Medicines, Inc. (納斯達克:PRAX)，一家臨床階段生物製藥公司，將基因洞察轉化爲針對中樞神經系統（CNS）障礙的療法，這些障礙以神經興奮與抑制不平衡爲特徵，今天宣佈美國食品藥品監督管理局（FDA）已授予relutrigine在德拉維特綜合症中的罕見兒科疾病指定（RPDD）。
譯文內容由第三人軟體翻譯。
