Expanded access: opening doors to personalized medicine for rare disease patients and patients with neurodegenerative diseases.
Expanded access: opening doors to personalized medicine for rare disease patients and patients with neurodegenerative diseases.
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扩大准入：为罕见疾病患者和神经退行性疾病患者打开个性化药物的大门。
DOI：
10.1111/febs.15529
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发表时间：
2021-03
期刊：
影响因子：
--
通讯作者：
Özdinler PH
中科院分区：
文献类型：
--
作者：
Özdinler PH
在神经退行性疾病中，一组选定的神经元群表现出早期易损性并经历进行性变性。大脑皮层的异质性和诊断为同一疾病的患者群体的异质性为开发有效和长期的治疗方案提供了许多挑战。目前，被认为患有“罕见”疾病的患者没有治愈的希望，许多神经退行性疾病进展迅速，没有任何有效的解决方案。然而，随着我们对疾病机制的理解的发展，我们开始意识到疾病之间的界限并不像以前认为的那样明显。有许多患者由于共同的潜在原因和机制而发病。随着药物研发工作的推进，很明显，我们将不得不把注意力从寻找治愈疾病的方法转移到寻找导致疾病的细胞机制的解决方案上，这样患者就可以通过基于机制的策略来治疗。这种模式的转变将为神经退行性疾病患者以及诊断为罕见疾病的患者的个性化医疗方法奠定基础。
In neurodegenerative diseases a select set of neuron population displays early vulnerability and undergo progressive degeneration. The heterogeneity of the cerebral cortex and the heterogeneity of patient populations diagnosed with the same disease offer many challenges for developing effective and long-term treatment options. Currently, patients who are considered to have a “rare” disease are left with no hopes for cure, and many of the neurodegenerative diseases progress fast without any effective solutions. However, as our understanding of disease mechanisms evolve, we begin to realize that the boundaries between diseases are not as sharp as once believed. There are many patients who develop disease due to common underlying causes and mechanisms. As we move forward with drug discovery effort, it becomes obvious that we will have to shift our focus from finding a cure for a disease, to finding solutions to the disease causing cellular mechanisms so that patients can be treated by mechanism-based strategies. This paradigm shift will lay the foundation for personalized medicine approaches for neurodegenerative disease patients as well as patients diagnosed with a rare disease.
影响因子：
1.5
作者：
Jarow, Jonathan P.;Lemery, Steven;Moscicki, Richard
通讯作者：
Moscicki, Richard
Edaravone如何对急性缺血性中风和肌萎缩性侧索硬化有效？
影响因子：
2.4
作者：
Watanabe K;Tanaka M;Yuki S;Hirai M;Yamamoto Y
通讯作者：
Yamamoto Y
影响因子：
4.6
作者：
Dervishi I;Gozutok O;Murnan K;Gautam M;Heller D;Bigio E;Ozdinler PH
通讯作者：
Ozdinler PH