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已上市SMA治療藥物對比-行業數據

已上市SMA治療藥物對比
已上市SMA治療藥物對比
細胞喪失支配。SMN1屬于常染色體隱性遺傳病，失活突變在正常人群的攜帶率為0.5-1%，只有父母 雙方均為致病基因攜帶者才會致病，新生兒患病率約為萬分之一。人類基因組中存在SMN2基因，與 SMN1幾乎相同，唯一的區別在于SMN2基因外顯子7的調控序列被異質核核糖蛋白（hnRNP）結合，在 2000 翻譯時缺少外顯子7，產生無功能且不穩定的蛋白產物（SMN2Δ7）。根據剩余的SMN2基因拷貝數量（1-4個），SMA可分為SMA1/2/3/4型
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已上市SMA治療藥物對比_第1頁
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已上市SMA治療藥物對比_第2頁
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已上市SMA治療藥物對比_第8頁
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已上市SMA治療藥物對比_第9頁
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已上市SMA治療藥物對比_第10頁
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已上市SMA治療藥物對比_第11頁
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已上市SMA治療藥物對比_第12頁
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已上市SMA治療藥物對比_第13頁
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已上市SMA治療藥物對比_第19頁
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已上市SMA治療藥物對比_第20頁
已上市SMA治療藥物對比_第21頁
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已上市SMA治療藥物對比_第22頁
已上市SMA治療藥物對比_第23頁
已上市SMA治療藥物對比_第24頁
已上市SMA治療藥物對比_第25頁
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已上市SMA治療藥物對比_第26頁
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已上市SMA治療藥物對比_第27頁
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已上市SMA治療藥物對比_第28頁
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已上市SMA治療藥物對比_第29頁
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已上市SMA治療藥物對比_第30頁
已上市SMA治療藥物對比_第31頁
已上市SMA治療藥物對比_第32頁
已上市SMA治療藥物對比_第33頁
已上市SMA治療藥物對比_第34頁
已上市SMA治療藥物對比_第35頁
已上市SMA治療藥物對比_第36頁
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已上市SMA治療藥物對比_第37頁
已上市SMA治療藥物對比_第38頁
已上市SMA治療藥物對比_第39頁
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已上市SMA治療藥物對比_第40頁
已上市SMA治療藥物對比_第41頁
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已上市SMA治療藥物對比_第42頁
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已上市SMA治療藥物對比_第43頁
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已上市SMA治療藥物對比_第44頁
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已上市SMA治療藥物對比_第45頁
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已上市SMA治療藥物對比_第46頁
所屬報告： 醫藥行業創新藥盤點系列報告（17）：引領現代制藥的第三波浪潮小核酸藥物帶來全新治療范式-230326（60頁）.pdf
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