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首页> 外文期刊>Drugs of today: Medicamentos de actualidad >Volanesorsen for treatment of patients with familial chylomicronemia syndrome.
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Volanesorsen for treatment of patients with familial chylomicronemia syndrome.

机译：用于治疗家族性Chylomronicronia综合征患者的valaneSorsen。

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Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder typically caused by mutations in genes for lipoprotein lipase (LPL), apolipoprotein C-II (Apo-CII), apolipoprotein A-V (Apo-AV), lipase maturation factor 1 (LMF1) and glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1 (GPI-HBP1). FCS is associated with severe morbidity that includes recurrent pancreatitis and other problems. Effective treatment to reliably prevent complications has been unavailable, so there is a quest to identify novel interventions to achieve sustained triglyceride lowering and prevention of pancreatitis. Apolipoprotein C-III (Apo-CIII) interferes with triglyceride clearance by blocking LPL and alternative pathways. Volanesorsen is an experimental antisense oligonucleotide that inhibits translation of Apo-CIII mRNA, thereby substantially lowering plasma levels of Apo-CIII and triglycerides. It is being developed for treatment of patients with FCS and refractory hypertriglyceridemia. Data from a variety of clinical trials have been very encouraging, with documentation of excellent triglyceride-lowering efficacy, but there have been concerns about the risk of drug-related thrombocytopenia and bleeding that contributed to the recent decision by the Food and Drug Administration (FDA) to not approve the drug for clinical use. Clinical trials testing the safety and efficacy of volanesorsen are ongoing, so there is hope that the drug ultimately will be approved and available for treatment of high-risk patients with FCS.
机译：家族性Chylomrronemia综合征（FCS）是一种稀有的常染色体隐性紊乱，通常由脂蛋白脂肪酶（LPL），载脂蛋白AV（APO-AV），脂肪酶成熟因子1（LMF1）中脂蛋白C-II（APO-CII）的突变引起和糖基膦酰氨基肌醇锚定的高密度脂蛋白结合蛋白1（GPI-HBP1）。 FCS与严重的发病率相关，包括复发性胰腺炎和其他问题。有效的治疗可靠地防止并发症不可用，因此有寻求识别新的干预措施，以实现持续的甘油三酯降低和预防胰腺炎。载脂蛋白C-III（APO-CIII）通过阻断LPL和替代途径干扰甘油三酯清除。 ValaneSorsen是一种实验反义寡核苷酸，其抑制Apo-CIII mRNA的翻译，从而显着降低了APO-CIII和甘油三酯的血浆水平。它正在开发用于治疗FCS和难治性高甘油血症患者。来自各种临床试验的数据一直非常令人鼓舞，具有优异的甘油三酯的疗效文件，但涉及毒药血小板减少症和出血的风险，这导致了食品和药物管理局最近的决定（FDA ）不批准临床用药。临床试验测试Valanesorsen的安全性和功效正在进行，因此希望药物最终将获得批准和可用于治疗FCS的高危患者。

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