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
Dravet 綜合徵是一種遺傳性發育和癲癇性腦病 (DEE)，通常由 SCN1A 的突變引起
This is the third Rare Pediatric Disease Designation for relutrigine, adding to those granted for SCN2A and SCN8A DEEs
這是瑞曲嗪獲得的第三個罕見兒科疾病稱號，此外還獲得了 SCN2A 和 SCN8A DEE 的認證
Praxis plans to initiate an all-DEE trial (EMERALD), inclusive of Dravet syndrome, in 1H2025
Praxis 計劃在 1H2025 中啓動一項包括 Dravet 綜合徵在內的 All-DEE 試驗 (EMERALD)
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（環球新聞專線）——Praxis Precision Medicines, Inc.（納斯達克股票代碼：PRAX）是一家臨床階段的生物製藥公司，該公司將遺傳見解轉化爲以神經元興奮抑制失衡爲特徵的中樞神經系統（CNS）疾病的療法的開發，今天宣佈，美國食品藥品監督管理局（FDA）已授予罕見兒科疾病認證（RPDD），以重新開發以神經元興奮抑制失衡爲特徵的中樞神經系統（CNS）疾病的療法。Dravet 綜合徵中的三嗪。
