通过临床试验的起起伏伏来支持亨廷顿舞蹈症家庭。

IF 2.1 Q3 NEUROSCIENCES Journal of Huntington's disease Pub Date : 2023-01-01 DOI:10.3233/JHD-230565
Kelly M Andrew, Leora M Fox
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引用次数: 0

摘要

近年来,亨廷顿舞蹈症(HD)研究领域一直动荡不安。三项HD临床试验,包括一种潜在的HD疾病修饰基因疗法的第一阶段3期试验,都在2021年3月暂停。2022年带来了更多的研究障碍和额外的试验终止。随着HD科学的进步和社区中更大规模的试验越来越频繁,HD家庭面临着临床研究很少导致新药上市的艰难现实。为了更好地了解亨廷顿舞蹈病社区如何为不断扩大的临床研究管道所带来的起伏做好准备,美国亨廷顿舞蹈病协会(HDSA)与亨廷顿舞蹈病患者参与联盟(HD- cope)的成员进行了交谈。这个由HDSA和加拿大亨廷顿舞蹈症协会(HSC)领导的全球倡导者组织与制药公司合作,确保在临床试验计划中代表HD的声音。通过这些对话,HDSA总结了如何通过临床研究过程在三个关键领域为HD社区提供最好的支持:参与、支持和教育。
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Supporting Huntington's Disease Families Through the Ups and Downs of Clinical Trials.

Recent years have been turbulent ones for the Huntington's disease (HD) community. Three clinical trials for HD, including the first Phase 3 trial of a potentially disease modifying genetic therapy for HD, were all brought to a halt in March of 2021. 2022 brought more study roadblocks and an additional trial termination. As HD science progresses and larger scale trials become more frequent in the community, HD families are faced with the difficult reality that clinical research rarely results in a new drug hitting the market. To better understand how the HD community can be prepared for the ups and downs that accompany an expanding clinical research pipeline, the Huntington's Disease Society of America (HDSA) spoke with members of the Huntington's Disease Coalition for Patient Engagement (HD-COPE). This group of global advocates led by HDSA and the Huntington's Society of Canada (HSC) collaborates with pharmaceutical companies to ensure that HD voices are represented in the planning of clinical trials. These conversations allowed HDSA to summarize how the HD community can be best supported through the clinical research process in three key areas: engagement, support, and education.

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来源期刊
CiteScore
4.80
自引率
9.70%
发文量
60
期刊最新文献
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