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首页> 外文期刊>Prescrire international >migalastat (galafold0) and Fabry disease No proof of efficacy and adverse effects still insufficiently known
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migalastat (galafold0) and Fabry disease No proof of efficacy and adverse effects still insufficiently known

机译：Migalastat（GalaFold0）和法布里疾病没有疗效证明和不良反应仍然不充分地知道

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Fabry disease is a rare genetic disorder caused by deficiency of the enzyme alpha-galactosidase A. Its most severe form, which generally affects males, presents in childhood or adolescence, and leads to cardiac, renal and cerebrovascular complications in young adulthood. The life expectancy of patients with severe disease is about 50 years. The benefit of enzyme replacement therapy with agalsidase alfa or beta is highly uncertain, due to the lack of proven efficacy based on clinical endpoints. Agalsidases are administered in hospital by intravenous infusion every 2 weeks. Migalastat (Galafold AmicusTherapeutics0) is claimed to stabilise some mutant forms of alpha-galactosidase A and thereby restore enzyme activity. It is taken orally every other day. Clinical evaluation of migalastat in patients with Fabry disease is based on two randomised trials: a 6-month double-blind placebo-controlled trial in 67 patients and an 18-month non-blinded trial versus agalsidase in 53 patients.The results of these trials showed no tangible difference between the groups in terms of the symptoms or clinical complications of the disease.
机译：法布里疾病是一种罕见的遗传疾病，由酶α-半乳糖苷酶A缺乏引起的遗传疾病。其最严重的形式，这通常会影响男性或青春期的雄性，并导致年轻成年期的心脏，肾和脑血管并发症。严重疾病患者的预期寿命约为50年。由于基于临床终点的缺乏经过验证的功效，酶替代疗法与Alfa Alfa或β的益处非常不确定。每2周通过静脉输注在医院中施用AgAlsidases。要求migalaStat（GalaFold Amicussteputics0）稳定一些突变形式的α-半乳糖苷酶A，从而恢复酶活性。它每隔一天都在口服。 Migalastat对法布里病患者的临床评价是基于两项随机试验：67名患者的6个月双盲安慰剂对照试验和53名患者的18个月的非蒙蔽试验。这些试验结果在疾病的症状或临床并发症方面没有在群体之间没有有形差异。

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    《Prescrire international》 |2018年第195期|共1页
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  • 中图分类 药学;
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