Designing a Plan for Drug Discovery in Rare Pediatric Neurodegenerative Disease

Danielle Kerkovich Ph.D. and; Amy Drew

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Designing a Plan for Drug Discovery in Rare Pediatric Neurodegenerative Disease

机译：设计罕见的小儿神经退行性疾病药物发现计划

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There are currently no cures for neurodegenerative diseases, including Batten disease, a rare and fatal disorder affecting young children. While researchers have made headway in preventing genetic disorders through preconception carrier screenings and have found potential drug targets, the gap between basic research and clinical treatment development remains. To overcome this gap, researchers in academia and the pharmaceutical industry, supported by government agencies and nonprofit institutions, must come together to share expertise and promote translational research.

机译：目前尚无治疗神经退行性疾病的方法，包括巴滕病，这是一种罕见的致命疾病，会影响幼儿。尽管研究人员已经通过孕前载体筛查在预防遗传疾病方面取得了进展，并且发现了潜在的药物靶标，但基础研究与临床治疗开发之间仍然存在差距。为了克服这一差距，在政府机构和非营利机构的支持下，学术界和制药业的研究人员必须聚集在一起，分享专业知识并促进转化研究。

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《Cerebrum: the Dana Forum on Brain Science》 |2011年第1期|共页
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Danielle Kerkovich Ph.D. and; Amy Drew;
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中图分类神经病学与精神病学;
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1. Drug Development for Neurodegenerative Diseases--Second Annual marcus evans Conference. Advances in drug development for NDD and expediting discovery through novel compounds and sound clinical trials. [J] . Morimoto B IDrugs: the investigational drugs journal . 2010,第7期

机译：神经退行性疾病的药物开发-第二届年度marcus evans会议。 NDD药物开发方面的进展以及通过新型化合物和可靠的临床试验加快发现的速度。
2. 3D human brain cell models: New frontiers in disease understanding and drug discovery for neurodegenerative diseases [J] . Paula Korhonen, Tarja Malm, Anthony R. White Neurochemistry International: The International Journal for the Rapid Publication of Critical Reviews, Preliminary and Original Research Communications in Neurochemistry . 2018,第期

机译：3D人脑细胞模型：疾病理解的新边界和神经变性疾病的药物发现
3. Induced Pluripotent Stem Cells for Disease Modeling and Drug Discovery in Neurodegenerative Diseases [J] . Cao Lei, Tan Lan, Jiang Teng, Molecular Neurobiology . 2015,第1期

机译：诱导多能干细胞用于神经退行性疾病的疾病建模和药物发现
4. An innovative framework for rare neurodegenerative diseases analysis [C] . Bevilacqua Vitoantonio, Grimaldi Gianluca, Semeraro Davide, IEEE International Symposium on Medical Measurements and Applications . 2015

机译：罕见神经退行性疾病分析的创新框架
5. Discovery of novel genes that cause rare pediatric renal diseases using whole-exome sequencing [D] . Lemaire, Mathieu. 2015

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6. Designing a Plan for Drug Discovery in Rare Pediatric Neurodegenerative Disease [O] . Danielle Kerkovich, Amy Drew 2011

机译：设计罕见的小儿神经退行性疾病药物发现计划
7. Curcumin as Scaffold for Drug Discovery against Neurodegenerative Diseases [O] . Filippa Lo Cascio, Paola Marzullo, Rakez Kayed, 2021

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Designing a Plan for Drug Discovery in Rare Pediatric Neurodegenerative Disease

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