Tackling the Challenges of Product Development Through a Collaborative Rare Disease Network: The Foundation Fighting Blindness Consortium.
Tackling the Challenges of Product Development Through a Collaborative Rare Disease Network: The Foundation Fighting Blindness Consortium.
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DOI：
10.1167/tvst.10.4.23
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发表时间：
2021-04-01
影响因子：
3
通讯作者：
Foundation Fighting Blindness Consortium Investigator Group
中科院分区：
文献类型：
--
作者：
Durham TA;Duncan JL;Ayala AR;Birch DG;Cheetham JK;Ferris FL 3rd;Hoyng CB;Pennesi ME;Sahel JA;Foundation Fighting Blindness Consortium Investigator Group
美国抗盲基金会是一家501(c)(3)非营利组织，于2016年成立了一个国际遗传性视网膜疾病专家联盟，其使命是加速开发罕见的遗传性视网膜变性的治疗方法，如色素性视网膜炎、Stargardt病、Leber先天性黑内障、Usher综合征、脉络膜血症和色盲。该联盟通过评估新的结果测量、共享标准化研究方案和数据集以及传播研究结果来完成其使命。该网络在前三年建立了研究基础设施，包括39个全球研究地点，现在准备与工业界合作扩大其新疗法试验的基础设施。这种模式代表了一种克服罕见病治疗发展挑战的创新方法。
The Foundation Fighting Blindness, a 501(c)(3) nonprofit organization, established an international consortium of inherited retinal disease specialists in 2016, with a mission to accelerate the development of treatments for rare, inherited retinal degenerations, such as retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, Usher syndrome, choroideremia, and achromatopsia. The Consortium accomplishes its mission by evaluating novel outcome measures, sharing standardized study protocols and datasets, and disseminating findings. Having established research infrastructure in the first 3 years, including 39 global research sites, the network is now poised to expand its infrastructure for trials of new therapies in partnership with industry. This model represents an innovative approach to overcome challenges of therapeutic development for rare diseases.
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影响因子：
8.1
作者：
Schonbach, Etienne M.;Strauss, Rupert W.;Scholl, Hendrik P. N.
通讯作者：
Scholl, Hendrik P. N.
DOI：
10.1002/ajmg.c.31825
发表时间：
2020-09
期刊：
American journal of medical genetics. Part C, Seminars in medical genetics
影响因子：
--
作者：
Mansfield BC;Yerxa BR;Branham KH
通讯作者：
Branham KH
影响因子：
8.1
作者：
Hariri, Amir H.;Zhang, Hong Yang;Sadda, SriniVas R.
通讯作者：
Sadda, SriniVas R.
影响因子：
8.1
作者：
Strauss, Rupert W.;Kong, Xiangrong;Smith, Monika
通讯作者：
Smith, Monika
影响因子：
3
作者：
Duncan JL;Pierce EA;Laster AM;Daiger SP;Birch DG;Ash JD;Iannaccone A;Flannery JG;Sahel JA;Zack DJ;Zarbin MA;and the Foundation Fighting Blindness Scientific Advisory Board
通讯作者：
and the Foundation Fighting Blindness Scientific Advisory Board