文摘
摘要目的:确定安全、耐受性和药代动力学/药效学(PD)关系的risdiplam non-naive脊肌萎缩症(SMA)患者。
背景:Risdiplam (EVRYSDI®)是一个中央和周边的分布式、口腔运动神经元的生存2 (SMN2)pre-mRNA拼接修饰符被FDA批准用于治疗SMA患者,年龄≥2个月。
设计/方法:珠宝鱼(NCT03032172)是一个多中心、开放研究的日常risdiplam non-naive SMA患者(6 months-60岁入选标准入学)曾收到RG7800 (RO6885247) nusinersen (SPINRAZA®),olesoxime或onasemnogene abeparvovec-xioi (ZOLGENSMA®)。
结果:登记人口(N = 174)包括一个广泛的年龄(1-60年),SMA类型(1 - 3),SMN2复制数字(1 - 4)和运动功能(non-sitters /模特/步行者)。我们以前从173名患者提供安全数据(数据削弱:2020年7月31日)收到risdiplam 41个月。一名患者退出了研究基线;剩余的患者,13日之前收到RG7800, 76收到nusinersen, 70收到olesoxime和14收到onasemnogene abeparvovec。Risdiplam治疗导致快速和持续,倍增加SMN蛋白水平与基线相比(数据削弱:2020年6月1日),这是符合PD翻车鱼首次治疗的研究的数据类型2/3 SMA患者。没有导致戒断毒品安全发现报告任何病人在珠宝鱼。risdiplam的安全性符合安全性在首次治疗的病人。
我们将12个月安全、PD和探索性的从珠宝鱼功效数据(数据削弱:2021年1月29日)。
结论:珠宝鱼正在在欧洲和美国的网站,并将提供重要的数据安全,PD和探索性的效果在广大民众中risdiplam non-naive SMA患者。
披露:奇博士已经收到个人薪酬在5000 - 9999美元的范围为Genentec担任顾问。奇博士已经收到个人薪酬在5000 - 9999美元的范围为在科学咨询服务或数据安全监测委员会罗氏。已收到奇博士研究的机构罗氏公司的支持。奇博士的机构已经接到Avexis研究支持。已收到奇博士研究的机构支持生原体。已收到奇博士的研究机构国家卫生研究院的支持。奇博士已经收到相关出版物的出版版税卫生保健。克劳迪奥·布鲁诺已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会诺华。克劳迪奥·布鲁诺已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会罗氏。克劳迪奥·布鲁诺已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会Sarepta。 Tina Duong has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Tina Duong has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dirk Fischer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Hoffmann La Roche AG. The institution of Dirk Fischer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffmann La Roche AG. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avexis. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Avexis. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. The institution of Janbernd Kirschner has received research support from Roche. The institution of Janbernd Kirschner has received research support from Biogen. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis, Scholar Rock, Cytokinetics. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis. The institution of Eugenio Mercuri has received research support from biogen. Marianne Gerber has received personal compensation for serving as an employee of Roche. Marianne Gerber has received stock or an ownership interest from F Hoffmann La Roche . Dr. Gorni has received personal compensation for serving as an employee of Hoffman La Roche. Heidemarie Kletzl has received personal compensation for serving as an employee of Hoffmann-La Roche. Miss Carruthers has received personal compensation for serving as an employee of Roche. Miss Carruthers has received stock or an ownership interest from Roche. Carmen Martin has received personal compensation for serving as an employee of Roche Products Ltd. Carmen Martin has received stock or an ownership interest from F. Hoffmann-La Roche Ltd. Francis Warren has received personal compensation for serving as an employee of Roche. Francis Warren has received stock or an ownership interest from Roche. Mariacristina Scoto has nothing to disclose.
