Acadia Pharmaceuticals Presents New DAYBUE Clinical Data At The 2024 American Academy Of Neurology Annual Meeting
Acadia Pharmaceuticals Presents New DAYBUE Clinical Data At The 2024 American Academy Of Neurology Annual Meeting
Acadia Pharmaceuticals Inc. (NASDAQ:ACAD) today announced that two oral presentations featuring DAYBUE (trofinetide) findings in Rett syndrome will be presented at the American Academy of Neurology (AAN) 2024 annual meeting, being held this week in Denver, CO.
阿卡迪亚制药公司（纳斯达克股票代码：ACAD）今天宣布，将在本周在科罗拉多州丹佛举行的美国神经病学会（AAN）2024年年会上发表两份口头报告，介绍DAYBUE（曲非替德）在雷特综合征中的发现。
The presentations include outcomes from the DAFFODIL, a Phase 2/3 open-label study evaluating the safety, tolerability and exploratory efficacy of DAYBUE in girls aged two to four living with Rett syndrome, as well as findings from exit interviews with caregivers of individuals living with Rett syndrome who participated in the LAVENDER, LILAC, LILAC-2 and DAFFODIL studies.
这些演讲包括DAFFODIL的结果，这是一项2/3期开放标签研究，评估了DAYBUE对两至四岁患有雷特综合征的女孩的安全性、耐受性和探索性疗效，以及对参与薰衣草、丁香、LILAC-2 和水仙花研究的雷特综合征患者护理人员的退出访谈结果。
AAN Oral Presentations:
AAN 口头演讲：
- Oral Presentation 003/Abstract 3540: Trofinetide for the Treatment of Girls Aged Two to Four Years with Rett Syndrome: Final Results from the Open-label DAFFODIL Study, Wednesday, April 17 at 3:54pm MT
- Oral Presentation 008/Abstract 2877: Assessing Experiences with Trofinetide for Rett Syndrome: Interviews with Caregivers of Patients in Open-label Studies, Wednesday, April 17 at 4:54pm MT
- 口头陈述 003/Abstract 3540：Trofinetide 用于治疗患有雷特综合症的两到四岁女孩：开放标签 DAFFODIL 研究的最终结果，美国东部时间 4 月 17 日星期三下午 3:54
- 口头报告 008/Abstract 2877：评估Trofinetide治疗雷特综合征的经验：对开放标签研究中患者护理人员的访谈，美国东部标准时间4月17日星期三下午 4:54
About Rett Syndrome
关于雷特综合症
Rett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and affects approximately 6,000 to 9,000 patients in the U.S., with approximately 5,000 patients currently diagnosed according to an analysis of healthcare claims data.1-4 A child with Rett syndrome exhibits an early period of apparently normal development until six to 18 months, when their skills seem to slow down or stagnate. This is typically followed by a duration of regression when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those living with Rett may continue to experience a stage of motor deterioration which can last the rest of the patient's life.5 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5 In preclinical studies, deficiency in MeCP2 function has been shown to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.5-7
雷特综合征是一种罕见的复杂神经发育障碍，可能分四个阶段发生，影响美国约6,000至9,000名患者，根据对医疗索赔数据的分析，目前约有5,000名患者被诊断出来。1-4 患有雷特综合征的儿童表现出明显正常的早期发育期，直到六到十八个月，那时他们的技能似乎减缓或停滞不前。当孩子失去获得的沟通技巧和有针对性的用手时，通常会有一段时间的回归。然后，孩子可能会经历一个停滞期，在此期间，他们的认知兴趣略有恢复，但身体动作仍然严重减弱。随着年龄的增长，患有Rett的患者可能会继续经历运动衰退阶段，该阶段可能会持续患者的余生。5 雷特综合征通常由Rett的基因突变引起 MECP2 gene.5 在临床前研究中，meCP2 功能的缺乏已被证明会导致突触沟通受损，突触功能的缺陷可能与 Rett 表现有关。5-7
Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.8 Most Rett patients typically live into adulthood and require round-the-clock care.2,9
雷特综合征的症状还可能包括形成手部刻板印象，例如拧手和拍手以及步态异常。8 大多数雷特患者通常活到成年，需要全天候护理。2,9
About DAYBUE (trofinetide)
关于 DAYBUE（trofinetide）
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. Trofinetide is thought to enhance neuronal synaptic function and morphology. This hypothesis is supported by findings from studies of trofinetide in a methyl-CpG-binding protein 2 gene (Mecp2) mouse model of Rett syndrome, in which increased branching of the dendrites that form synapses and synaptic plasticity signals were observed.10
Trofinetide 是胰岛素样生长因子 1 的 N 末端三肽的合成类似物。trofinetide对雷特综合征患者发挥治疗作用的机制尚不清楚。Trofinetide被认为可以增强神经元突触功能和形态。对甲基-CPG结合蛋白2基因中的曲非内肽的研究结果支持了这一假设（Mecp2) 雷特综合征的小鼠模型，在该模型中观察到形成突触和突触可塑性信号的树突分支增多。10
译文内容由第三方软件翻译。