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Outline
Transthyretin amyloidosis (ATTR) disease is caused by abnormal folding of transthyretin (TTR) in tissues, which mainly involves the peripheral nervous system and heart, progressively aggravates to even be life-threatening. Currently, 3 TTR-targeted therapeutic drugs have been marketed in the United States and Europe. The information from these drugs' nonclinical pharmacology and toxicology studies has important implications for 3 clinical effectiveness and safety. This article summarized the key points of the nonclinical studies of these three TTR-targeted therapeutic drugs and elaborated the focus of nonclinical study and evaluation of TTR-targeted therapeutic drugs for ATTR disease from the aspects of nonclinical effectiveness and nonclinical safety to provide information for new drug development in China.
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| 科 Family | 属数 Number of genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) | 属 Genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) |
|---|---|---|---|---|---|---|
| 鹅膏菌科Amanitaceae | 2 | 11 | 5.26 | 鹅膏菌属 Amanita | 10 | 4.78 |
| 小菇科 Mycenaceae | 2 | 12 | 5.74 | 丝盖伞属 Inocybe | 5 | 2.39 |
| 多孔菌科 Polyporaceae | 8 | 14 | 6.70 | 蜡蘑属 Laccaria | 5 | 2.39 |
| 红菇科 Russulaceae | 3 | 23 | 11.00 | 小皮伞属 Marasmius | 6 | 2.87 |
| 小菇属 Mycena | 11 | 5.26 | ||||
| 光柄菇属 Pluteus | 5 | 2.39 | ||||
| 红菇属 Russula | 17 | 8.13 | ||||
| 栓菌属 Trametes | 5 | 2.39 |
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